New FDA Approvals

Use AI to Speed up Your Work Flow Ethically and Securely. There are lots of courses on AI out there, but this one is specific only to medical writing and editing.  Access the AIMWE course waitlist. https://www.learnamastyle.com/waitlist/ Leqselvi for Alopecia Areata: The FDA has approved deuruxolitinib (Leqselvi) as a first-line treatment for adults with moderate to severe alopecia areata (AA). Developed by Sun Pharma, this oral selective inhibitor of Janus kinases JAK1 and JAK2 is typically dosed at 8 mg twice daily. The approval, based on the THRIVE-AA1 and THRIVE-AA2 trials, marks an important addition to limited treatment options for AA, which has significant psychological impacts. Femlyv Dissolvable Birth Control: The FDA has approved norethindrone acetate and ethinyl estradiol (Femlyv) as an oral dissolvable birth control pill, expanding access for those who have difficulty swallowing. This form of hormonal birth control, in use since 1968 as a swallowable tablet, also helps manage conditions like endometriosis and PMDD. The approval, based on a study of 743 women, offers a new option with common side effects like headache and nausea. Brineura for Batten Disease: The FDA has expanded the indication for cerliponase alfa (Brineura) to treat neuronal ceroid lipofuscinosis type 2 (CLN2 disease) in children under 3 years of age. Developed by BioMarin Pharmaceutical, this enzyme replacement therapy, initially approved in 2017, is administered by infusion into the brain. The expanded approval was based on a phase 2 trial showing reduced motor function decline and delayed disease onset. Erzofri for Schizophrenia: The FDA has approved paliperidone palmitate (Erzofri) extended-release injectable suspension for treating schizophrenia and schizoaffective disorder in adults. This long-acting injectable (LAI) antipsychotic, administered once a month, improves patient adherence by reducing dosing frequency. Developed by Luye Pharma Group, it was granted a U.S. patent in 2023 and approved under the 505(b)(2) pathway. Eque-cel for MS: The FDA has approved the IND application for equecabtagene autoleucel (Eque-cel) for treating multiple sclerosis (MS). Developed by IASO Biotechnology, this chimeric antigen receptor autologous T cell injection showed promising efficacy in six autoimmune diseases in a Chinese trial. MS, affecting 3.07 million people worldwide, is characterized by CNS demyelination and neuronal injury, with Eque-cel offering a new treatment option. Spravato for TRD: Johnson & Johnson has submitted an sNDA for esketamine (Spravato) as a monotherapy for adults with treatment-resistant depression (TRD). Already approved in combination with an oral antidepressant, esketamine is an NMDA receptor antagonist that rapidly alleviates depressive symptoms. The submission is based on Phase 4 TRD4005 study results, showing rapid improvement in depression scores with a consistent safety profile. ZW191 Anti-Tumor Agent: The FDA has cleared the IND application for ZW191, a novel antibody-drug conjugate (ADC) targeting folate receptor alpha (FR⍺) in cancers like gynecologic and NSCLC. Developed by Zymeworks, this TOPO1i ADC uses a novel payload, ZD06519, showing robust antitumor activity and a tolerable safety profile in preclinical models. Clinical development of ZW191 is planned to begin in the second half of 2024.

Visit learnAMAstyle.com to uplevel your writing and editing skills with free downloads on medical writing and editing. The FDA has approved vonoprazan (Voquenza) for treating non-erosive gastroesophageal reflux disease (GERD) in adults. Vonoprazan is an oral potassium-competitive acid blocker (PCAB) already approved for erosive esophagitis and Helicobacter pylori infection. PCABs are a new class of medicines that inhibit stomach acid secretion. Non-erosive GERD is the most common form of GERD, affecting around 45 million US adults, with about 15 million treated with prescription medication annually. Many patients remain dissatisfied with existing treatments, experiencing symptoms that affect their quality of life. The approval of vonoprazan was based on the Phalcon-Nerd-301 study, a Phase 3 trial showing it significantly reduced heartburn episodes and provided more heartburn-free days and nights compared to placebo. The trial included 772 adults with frequent heartburn, demonstrating vonoprazan's effectiveness over placebo. The FDA has granted fast track designation to ADI-270 for metastatic or advanced clear cell renal cell carcinoma (ccRCC) in patients who previously received an immune checkpoint inhibitor and a VEGF inhibitor. ADI-270 is a CD70-targeted gamma delta CAR T-cell therapy designed to enhance resilience to the tumor microenvironment. A phase 1/2 trial will evaluate ADI-270 in patients with confirmed ccRCC who have been previously treated. The primary endpoints are the incidence of dose-limiting toxicities and the proportion of treatment-emergent adverse effects. The FDA has granted Fast Track and Regenerative Medicine Advanced Therapeutic (RMAT) designations to Lomecel-B, an investigational MSC therapy for Alzheimer's disease (AD). Lomecel-B aims to address neurodegeneration in AD and showed positive results in the CLEAR MIND Phase 2a trial. Visit learnAMAstyle.com to uplevel your writing and editing skills with free downloads on medical writing and editing.

Visit learnAMAstyle.com for free downloads in writing in medicine and the life sciences.    The FDA has approved blinatumomab (Blincyto) for treating adult and pediatric patients one month or older with CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (B-ALL) in the consolidation phase. This bispecific T-cell engager (BiTE®) therapy targets CD19 surface antigens on B cells, allowing T cells to recognize and eliminate malignant B cells. The approval was based on the Phase 3 E1910 trial showing improved overall survival with blinatumomab plus chemotherapy compared to chemotherapy alone.   The FDA has approved roflumilast cream 0.15% (Zorvye) for treating mild to moderate atopic dermatitis (AD) in patients aged 6 years and older. This steroid-free, once-daily topical treatment is a selective, highly potent phosphodiesterase 4 (PDE4) inhibitor designed for long-term disease control. The approval was based on phase 3 studies showing significant improvements in disease clearance, itch reduction, and overall skin condition compared to the vehicle group.   The FDA has approved a new 6mg single-dose prefilled syringe for faricimab-svoa (Vabysmo), facilitating easier administration for neovascular (wet) age-related macular degeneration (AMD), diabetic macular edema (DME), and macular edema following retinal vein occlusion (RVO). Faricimab-svoa is a VEGF and Ang-2 inhibitor, now available in a ready-to-use format. This approval provides an alternative to the existing single-dose vial and is granted to Genentech.   The FDA is set to decide on OX124, a high-dose naloxone nasal rescue medication designed to reverse opioid overdoses, with the PDUFA date set for July 15, 2024. Developed by Orexo, OX124 offers rapid absorption, high bioavailability, and enhanced stability, addressing the critical need for potent rescue medications due to the high rate of synthetic opioid overdoses. If approved, OX124's launch is anticipated later in 2024.   The FDA accepted an NDA for vonoprazan (Voquezna) tablets for treating heartburn associated with Non-Erosive gastroesophageal reflux disease (GERD) in adults, with the PDUFA date set for July 19, 2024. Vonoprazan is a potassium-competitive acid blocker (PCAB) offering an alternative to traditional proton pump inhibitors (PPIs). Non-Erosive GERD, affecting 38 million U.S. adults, is characterized by reflux-related symptoms without esophageal mucosal erosions, impacting quality of life with symptoms like heartburn and chest pain.  

Visit learnamastyle.com for free downloads and free courses related to writing in medicine and science.  - The FDA has approved donanemab (Kisunla) for early symptomatic Alzheimer's disease, making it the third drug in a new class aimed at slowing cognitive decline in early-stage patients. Eli Lilly will make the drug available within weeks following this approval. Donanemab is an anti-amyloid monoclonal antibody that targets amyloid plaques in the brain, a key feature of Alzheimer's.  - The FDA approval was based on clinical trials showing significant benefits in slowing cognitive decline despite concerns about long-term safety. The advisory committee voted unanimously in favor of the drug, with the FDA granting the approval to Eli Lilly. - The FDA has approved bedaquiline (Sirturo) for treating multidrug-resistant pulmonary tuberculosis (TB) in adults and children aged five and above. Sirturo initially received accelerated FDA clearance for adults in 2012, with later label expansions for younger patients. Bedaquiline, the first TB drug with a new action mechanism in over 40 years, inhibits mycobacterial ATP synthase, essential for energy production in TB bacteria.. - The FDA is nearing a decision on KarXT, a novel antipsychotic for schizophrenia developed by Karuna Therapeutics. KarXT has shown efficacy in reducing symptom severity in clinical trials, with a favorable side effect profile compared to older antipsychotics. Concerns about insurance coverage exist due to the availability of cheaper generics, but a decision is expected by September 26. - MDMA-assisted therapy for PTSD, developed by Lykos Therapeutics, has shown significant symptom reduction in clinical trials but faces approval challenges. Despite positive trial results, the FDA advisory panel voted against recommending the drug, citing safety concerns. The FDA's final decision is expected by August 11.  - Seladelpar, under consideration for primary biliary cholangitis (PBC), is a drug Gilead recently acquired through the purchase of CymaBay. If approved by August 14, it would expand Gilead's liver disease portfolio. Clinical trials have shown seladelpar to be effective in reducing the itching associated with PBC. - TransCon PTH by Ascendis Pharma is a treatment for hypoparathyroidism, designed to replace parathyroid hormone and help patients achieve normal calcium levels. Despite manufacturing concerns delaying the FDA's decision, the drug has already been approved in Europe and the UK. A final FDA decision is expected by August 14.  - Afami-cel, a T-cell receptor therapy for synovial sarcoma developed by Adaptimmune Therapeutics, awaits FDA approval by August 4. The therapy targets the MAGE A4 cancer target and is designed as a single-dose treatment. Clinical trial data supporting the application showed efficacy in treating advanced synovial sarcoma.

Do you work for a CME company that needs urgent medical writing assistance? We can help. Visit nascentmc.com - The FDA has approved ensifentrine (Ohtuvayre) as an inhaled maintenance therapy for adults with COPD. Ohtuvayre is the first new mechanism of action for COPD treatment in over two decades, offering anti-inflammatory and bronchodilatory effects to improve lung function and reduce symptoms without the side effects of inhaled corticosteroids. The approval was based on Phase 3 trials showing significant improvement in lung function and fewer exacerbations, granted to Verona Pharma. - The FDA has approved thiotepa (Tepylute) for breast and ovarian cancer treatment. This new ready-to-dilute formulation saves preparation time and reduces risks, offering benefits like less-invasive treatment options. Breast cancer affects 1 in 8 women, while ovarian cancer has poorer outcomes due to late detection; the approval emphasizes Tepylute's ease of preparation and was granted to Amneal Pharmaceuticals. - The FDA has approved epcoritamab-bysp (EPKINLY) for adults with relapsed or refractory follicular lymphoma (FL) after two or more lines of prior therapy. EPKINLY is the first T-cell engaging bispecific antibody for this indication, providing a new option for patients with limited treatment choices. The approval was based on the EPCORE® NHL-1 clinical trial, showing a high overall response rate, granted to AbbVie. - The FDA has approved sofpironium topical gel (Sofdra) for treating primary axillary hyperhidrosis in adults and children aged 9 and older. Sofdra, an anticholinergic agent, reduces sweat production and significantly improves quality of life. The approval, based on two phase 3 studies showing significant improvement in sweat production, was granted to Botanix Pharmaceuticals, with expected availability in late 2024. - Nascent Medical offers exceptional medical writing support with skilled MD- and PhD-level writers specializing in needs assessments, manuscripts, slide decks, and more. Visit nascentmc.com for excellent medical writing assistance.  - The FDA has approved pitolisant (Wakix) for treating excessive daytime sleepiness (EDS) in pediatric patients with narcolepsy. Pitolisant, a first-in-class treatment targeting the histamine system, was previously approved for adults with narcolepsy. The approval, based on a Phase 3 study, was granted to Harmony Biosciences. - The FDA has approved crovalimab-akkz (PiaSky) for treating paroxysmal nocturnal hemoglobinuria (PNH) in patients aged 13 and older. Crovalimab, a complement C5 inhibitor, provides sustained complement inhibition through low-dose administration. The approval, based on efficacy in maintaining complement inhibition, was granted to Genentech, Inc. - The FDA has approved efgartigimod alfa and hyaluronidase-qvfc (Vyvgart Hytrulo) for treating chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. Vyvgart Hytrulo, previously approved for generalized myasthenia gravis, demonstrated effectiveness in a two-stage study showing significant improvement in patient outcomes. The approval was granted to Argenx.  - The FDA has granted fast track designation to IBI343 for treating advanced pancreatic ductal adenocarcinoma (PDAC) that has relapsed or is refractory to one prior therapy. IBI343 is a monoclonal antibody-drug conjugate targeting CLDN18.2-expressing tumor cells. The designation, based on a phase 1 trial showing a 40% overall response rate, was granted to Innovent Biologics.

Visit nascentmc.com for the full writup of this episode and medical writing assistance.  Visit learnamastyle.com for free downloads directed towards medical writing and editing. • The FDA has approved the 21-valent pneumococcal conjugate vaccine, CAPVAXIVE™ (Merck), for the prevention of invasive disease and pneumonia in adults aged 18 years and older caused by 21 Streptococcus pneumoniae serotypes. Capvaxive includes eight serotypes not covered by other pneumococcal vaccines, addressing approximately 27% of IPD cases in adults aged 50 and older, and 30% in adults aged 65 and older, based on CDC data from 2018-2021. The approval follows an FDA Priority Review and is based on immune responses measured in the Phase 3 STRIDE-3 trial, with continued approval contingent upon verification of clinical benefit in a confirmatory trial.  • The FDA has approved delandistrogene moxeparvovec-rokl (Elevidys) for Duchenne muscular dystrophy (DMD) in ambulatory individuals aged 4 and older with a confirmed mutation in the DMD gene, as well as granting accelerated approval for non-ambulatory individuals. Elevidys, a one-time intravenous gene therapy, delivers a working copy of the DMD gene to address the muscle degeneration caused by mutations in this gene. The approvals are based on findings from a confirmatory trial that, while not meeting its primary endpoint, showed success in several secondary measures, with the Phase 3 ENVISION study underway to serve as a postmarketing requirement. • The FDA has approved adagrasib (Krazati) plus cetuximab for adults with KRAS G12C-mutated locally advanced or metastatic colorectal cancer (CRC) who have received prior treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy. Adagrasib targets the KRAS G12C mutation, a common driver mutation in several cancers including colorectal cancer, while cetuximab enhances its antitumor activity. The approval was based on findings from the KRYSTAL-1 trial, which demonstrated a confirmed overall response rate (ORR) of 34% and a median duration of response (DOR) of 5.8 months. • The FDA has approved pembrolizumab (Keytruda) in combination with carboplatin and paclitaxel, followed by pembrolizumab monotherapy, to treat primary advanced or recurrent endometrial carcinoma in adults, marking the third endometrial carcinoma indication for Keytruda in the US. Keytruda enhances the body's immune response against tumor cells by blocking the interaction between PD-1 and its ligands, PD-L1 and PD-L2. The approval is based on results from the phase 3 KEYNOTE-868 clinical trial, which demonstrated significant improvements in progression-free survival for patients treated with Keytruda plus chemotherapy compared to those receiving a placebo with chemotherapy.  • The FDA has approved risankizumab-rzaa (Skyrizi) for the treatment of moderately to severely active ulcerative colitis in adults, making it the first specific anti–interleukin 23 monoclonal antibody indicated for both ulcerative colitis and moderate to severe Crohn's disease. Risankizumab-rzaa inhibits interleukin-23 (IL-23), a cytokine involved in inflammatory and immune responses, thereby reducing inflammation. The approval is based on data from two phase 3 clinical trials, INSPIRE and COMMAND, which demonstrated the achievement of clinical remission and endoscopic improvement. • A supplemental Biologics License Application (sBLA) has been submitted for guselkumab (Tremfya) for the treatment of adults with moderately to severely active Crohn's disease. Guselkumab, a fully-human, dual-acting monoclonal antibody that blocks IL-23 and binds to CD64, was previously approved for moderate-to-severe plaque psoriasis and active psoriatic arthritis. Support for the BLA is based on findings from the Phase 3 GALAXI and GRAVITI clinical trials.

Check out Nascentmc.com for medical writing assistance. visit learnAMAstyle.com for free downloads on medical writing and editing Adbry for Atopic Dermatitis The FDA has approved tralokinumab-ldrm (Adbry) as a 300 mg single-dose autoinjector for moderate-to-severe atopic dermatitis (AD) in adults, offering a more convenient delivery method. Adbry, which inhibits IL-13, was previously approved for adults in December 2021 and for pediatric patients aged 12 and older in December 2023. The approval was granted to LEO Pharma Inc.  Augtyro for NTRK Tumors The FDA has approved repotrectinib (Augtyro) for treating solid tumors with NTRK gene fusions in patients aged 12 and older, based on Phase 1/2 trials showing significant response rates in both TKI-naïve and previously treated patients. The approval was granted to Bristol Myers Squibb, with additional clinical data required to confirm safety and efficacy. Donanemab for Alzheimer's FDA advisors unanimously recommended the approval of donanemab for Alzheimer's disease, emphasizing its efficacy in slowing early-stage disease and manageable risks. Donanemab, targeting amyloid plaques, offers potential advantages over Leqembi with monthly infusions. The FDA decision is expected soon. Iqirvo for Primary Biliary Cholangitis The FDA granted accelerated approval to elafibranor (Iqirvo) for primary biliary cholangitis (PBC) to be used with ursodeoxycholic acid or as monotherapy. Elafibranor targets PPAR-α and PPAR-δ, with Phase 2 trials showing significant biochemical responses. The approval was granted to GENFIT and Ipsen.  Retevmo in Thyroid Cancer The FDA granted full approval to selpercatinib (Retevmo) for advanced or metastatic RET fusion–positive thyroid cancer in patients aged 2 years and older, based on the LIBRETTO-001 trial showing high response rates. The approval was granted to Eli Lilly and Company. OTC Continuous Glucose Monitors The FDA approved Abbott Laboratories' continuous glucose monitoring systems, Libre Rio and Lingo, for over-the-counter use. Libre Rio is for Type 2 diabetes patients not on insulin, while Lingo targets non-diabetic consumers for health improvement. These systems provide real-time glucose monitoring via a smartphone app. Check out Nascentmc.com for medical writing assistance.visit learnAMAstyle.com for free downloads on medical writing and editing  

Nascentmc.com for medical writing assistance for your company. Visit learnAMAstyle.com for free downloads on medical writing and editing Klisyri for Actinic Keratosis: The FDA has approved tirbanibulin (Klisyri) for treating actinic keratosis (AK) on larger areas of the face or scalp, up to 100 cm². Previously approved for areas up to 25 cm² in December 2020, tirbanibulin is now available in a 350 mg package size for a 5-day topical treatment. Actinic keratosis is the most common pre-cancerous dermatological condition in the US, and this approval was based on a Phase 3 clinical study showing consistent safety and effectiveness for larger treatment areas. MDMA for PTSD: An FDA advisory panel voted 10-1 against endorsing MDMA for PTSD treatment due to flawed study data, questionable research practices, and significant drug risks. The FDA will make its final decision on August 11, but the panel's negative feedback might influence a denial. This decision could impact the approval and acceptance of other psychedelics like LSD and psilocybin for medical use. Arexvy for RSV: The FDA has approved the RSV vaccine AREXVY for preventing RSV lower respiratory tract disease in adults aged 50-59 at increased risk. Initially approved in May 2023 for adults aged 60 and older, AREXVY combines a recombinant RSV glycoprotein with an adjuvant. The approval followed a Phase III trial showing the vaccine's immune response and safety in the targeted age group. Rytelo for MDS: The FDA has approved imetelstat (RYTELO) for treating low- to intermediate-1 risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia. Imetelstat, a telomerase inhibitor, demonstrated significant improvements in transfusion independence and hemoglobin levels in the IMerge Phase 3 trial. The approval was granted to Geron Corporation. Firdapse for LEMS: The FDA has approved an increased dose of amifampridine (Firdapse) from 80 mg to 100 mg for treating Lambert-Eaton myasthenic syndrome (LEMS) in adults and pediatric patients over 45 kg. Initially approved in 2018 for adults, and expanded to include children as young as 6 in 2022, the increased dose approval was based on clinical trials showing significant improvements in muscle strength. The approval was granted to Catalyst Pharmaceuticals. Nascentmc.com for medical writing assistance for your company. Visit learnAMAstyle.com for free downloads on medical writing and editing

Go here for complete show notes.   Visit learnAMAstyle.com for free downloads on medical writing and editing Nascentmc.com for medical writing assistance for your company.   mRESVIA for Lower Respiratory Tract Disease The FDA approved mRNA-1345 (mRESVIA) for preventing RSV-caused lower respiratory tract disease in adults 60 and older, based on Phase 3 trial results showing 83.7% efficacy and no serious safety concerns.  Breyanzi for R/R MCL The FDA approved lisocabtagene maraleucel (Breyanzi) for treating relapsed or refractory mantle cell lymphoma (MCL) after at least two prior therapies, demonstrating an 85.3% overall response rate in trials. Sarclisa for ND MM The FDA accepted the sBLA for isatuximab (Sarclisa) combined with VRd for treating transplant-ineligible newly diagnosed multiple myeloma, showing significant improvement in progression-free survival. Onyda XR for ADHD The FDA approved clonidine hydrochloride (Onyda XR) for treating ADHD in pediatric patients aged 6 and older, as a liquid nonstimulant medication with common side effects like somnolence and sedation. Bkemv for PNH and aHUS The FDA approved eculizumab-aeeb (Bkemv) as the first interchangeable biosimilar to eculizumab for treating PNH and atypical hemolytic uremic syndrome, requiring meningococcal vaccination prior to use. Austedo in Huntington's The FDA approved a new once-daily tablet option for deutetrabenazine (Austedo XR) for tardive dyskinesia and chorea in Huntington's disease, enhancing treatment flexibility and adherence based on long-term safety and effectiveness data.  

            For medical writing assistance, please visit nascentmc.com learnAMAstyle.com for the AI in Medical Writing and Editing: Blueprint for Success in a Changing World course to assist with implementing AI in medical writing.  •    Imdelltra for EC-SCLC: The FDA granted accelerated approval for tarlatamab-dlle (Imdelltra) for adults with extensive-stage small cell lung cancer (ES-SCLC) who progressed after platinum-based chemotherapy. Small cell lung cancer, accounting for 10-15% of all lung cancers, is aggressive with a median survival of 12 months post-therapy. Imdelltra, a first-in-class BiTE therapy targeting DLL3, showed a 40% objective response rate and a median overall survival of 14.3 months in the phase 2 DeLLphi-301 trial. •    Breyanzi for R/R LL: The FDA granted accelerated approval for lisocabtagene maraleucel (Breyanzi) for adults with relapsed or refractory follicular lymphoma after two or more systemic therapies. This CAR T-cell therapy, already approved for other B-cell malignancies, genetically modifies T cells to target tumor antigens. In the TRANSCEND FL trial, Breyanzi achieved a 95.7% overall response rate in 94 patients, with the median response duration not reached after 16.8 months. •    SNB-101 for SCLC: The FDA granted fast-track designation to SNB-101 for small cell lung cancer (SCLC). SNB-101, a polymer nanoparticle formulation of the antineoplastic agent SN-38, enhances lung-specific delivery and efficacy. Early data indicates improved tolerability and efficacy, with global phase 2 trials planned for various solid tumors including colon and gastric cancers, led by SN BioScience. •    Upstaza for AADC Deficiency: The FDA accepted the Biologics License Application for eladocagene exuparvovec (Upstaza), a gene therapy for aromatic L–amino acid decarboxylase (AADC) deficiency, with a Priority Review and a target date of November 13, 2024. Upstaza delivers the human DDC gene to the putamen, increasing AADC enzyme levels to restore dopamine production, showing significant neurological improvements in clinical trials. Approval was granted to PTC Therapeutics, Inc. •    Altuviiio for Hemophilia A Label Update: The FDA approved an updated label for Altuviiio (antihemophilic factor, recombinant), including phase 3 XTEND-Kids trial results. This high-sustained factor VIII replacement therapy, initially approved in 2023, offers prolonged bleed protection for pediatric patients under 12 years with hemophilia A. The updated label confirms Altuviiio's safety and effectiveness for both routine and on-demand treatment. •    Self-Collected Sample for Cervical Cancer Screening: The FDA approved the BD Onclarity™ HPV Assay for self-collected vaginal specimens for HPV testing when traditional cervical samples are not available. This assay, identifying six HPV strains, enhances screening access, especially for underserved populations with higher cervical cancer rates. Approval was granted to Becton Dickinson. •    Clinolipid Parenteral Nutrition: The FDA approved Clinolipid (Lipid Injectable Emulsion) for pediatric patients, including preterm and term neonates. Clinolipid, a mixed oil lipid emulsion providing essential fatty acids and calories, has been available for adults since 2019 and is now approved for all ages. Approval was granted to Baxter International Inc.

Visit NascentMC.com for medical writing assistance. Visit learnAMAstyle.com for information on AI in Medical Writing and Editing - **mRNA Anti-EBV Cancer Vaccine**: The novel mRNA therapeutic cancer vaccine WGc-043 has been approved for clinical trials by the FDA, targeting Epstein-Barr virus (EBV) related cancers such as nasopharyngeal carcinoma and natural killer T-cell lymphoma. It stimulates the immune system to respond against EBV and associated malignancies, showing superior efficacy and safety in preliminary trials. A phase 1 clinical trial is currently focusing on patients who have failed second-line therapies, aiming to determine the optimal dose and evaluate safety and efficacy metrics. - **BTX-9341 for Breast Cancer**: The FDA has approved the investigational new drug application for BTX-9341, a novel cyclin-dependent kinase (CDK) 4/6 bifunctional degrader, intended for hormone receptor-positive, HER2-negative breast cancer resistant to existing CDK4/6 inhibitors. This drug targets and degrades CDK4/6 proteins, crucial for cancer cell cycle regulation, aiming to overcome resistance to current treatments. A phase 1 trial will assess its safety, biological activity, and efficacy both as monotherapy and combined with fulvestrant. - **Nivolumab Hyaluronidase Formulation**: The FDA has accepted a Biologics License Application for a subcutaneous formulation of nivolumab co-formulated with recombinant human hyaluronidase, enhancing convenience by reducing administration time compared to the intravenous version. This application includes all previously approved solid tumor indications for nivolumab, with a PDUFA date set for February 28, 2025. The subcutaneous version aims to provide faster and easier administration, supported by data from the Phase 3 CheckMate-67T study. - **ColoSense Colorectal Cancer Screening Test**: The FDA has approved ColoSense, a noninvasive colorectal cancer screening test using multi-target stool RNA for adults aged 45 and older at average risk. ColoSense, distinct from traditional FOBT tests, uses RNA biomarkers to detect CRC with high sensitivity and has shown promising results in clinical trials, detecting 93% of CRC cases and 45% of advanced adenomas in average-risk individuals. This new test offers a significant improvement in sensitivity and specificity over existing methods. For the complete shownotes visit nascentmc.com/podcast

Please visit Nascentmc.com for medical writing assistance for your company. Visit learnAMAstyle.com for free downloads on medical writing and editing Ingrezza for Huntington's: The FDA has approved valbenazine (INGREZZA® SPRINKLE) capsules for adults with tardive dyskinesia and chorea associated with Huntington's disease. This new oral granules formulation provides an alternative for those with swallowing difficulties, maintaining the same dosage strengths as the original capsule form. Valbenazine is a selective vesicular monoamine transporter 2 (VMAT2) inhibitor designed to reduce uncontrollable movements by inhibiting dopamine release. Tivdak for Cervical Cancer: Tisotumab vedotin-tftv (TIVDAK) has been approved for recurrent or metastatic cervical cancer after chemotherapy failure. This approval was based on results from a Phase 3 trial showing a significant overall survival benefit compared to chemotherapy. The approval highlights a significant advancement in treating this aggressive form of cancer, granted to Pfizer and Genmab A/S. Xolremdi for WHIM: Mavorixafor (Xolremdi) has been approved for WHIM syndrome in patients 12 years and older to increase circulating neutrophils and lymphocytes. WHIM syndrome is a rare genetic disorder impairing immune function, and mavorixafor significantly improves cell counts and reduces infection risk. Approval was based on positive results from a double-blind trial and granted to X4 Pharmaceuticals. Esprit Stent for CLTI-BTK: The FDA has approved the Esprit™ BTK Everolimus Eluting Resorbable Scaffold System for chronic limb-threatening ischemia below-the-knee. This is the first approval of its kind in the U.S., offering an alternative to traditional balloon angioplasty. The approval was based on positive outcomes from the LIFE-BTK trial, showing significant disease progression reduction compared to standard care, granted to Abbott Laboratories.  

VIsit Nascentmc.com for medical writing assistance. Visit learnamastyle.com for freebies on medical writing and editing and ChatGPT. - Fasenra Pediatric Asthma Expansion: No details provided for this update. - Beqvez for Hemophilia B: FDA approved gene therapy, Beqvez, for adults with moderate to severe hemophilia B, which enables the production of clotting protein factor IX. This one-time treatment by Pfizer, derived from Spark Therapeutics, aims to replace frequent infusion therapies, demonstrating superior efficacy in a late-stage trial. - Anktiva for Bladder Cancer: Anktiva received FDA approval for treating BCG-unresponsive non-muscle invasive bladder cancer, enhancing NK and T cell proliferation. Based on a trial with 77 patients, it showed a 62% complete response rate, surpassing international clinical benchmarks, leading to its designation as a breakthrough therapy. - Pivya for UTI: FDA approved pivmecillinam (Pivya) for treating uncomplicated urinary tract infections caused by specific bacterial strains. This marks the first new antibiotic for such infections in the U.S. in over 20 years, backed by effective outcomes in three clinical trials. - Tovorafenib for Pediatric Low-Grade Glioma: Tovorafenib was approved for pediatric low-grade glioma patients with specific BRAF alterations, showing a 67% response rate in the FIREFLY-1 trial. It has been designated for accelerated approval due to its potential in treating these brain tumors. - Entyvio Maintenance for Crohn's Disease: Vedolizumab (Entyvio) received approval for subcutaneous administration as Crohn's disease maintenance therapy after initial intravenous induction. Supported by the VISIBLE 2 Study, it proved effective in maintaining clinical remission at 52 weeks. - Alecensa for NSCLC: Alecensa was approved as an adjuvant treatment post-tumor resection for ALK-positive non-small cell lung cancer. In the ALINA trial, it significantly extended disease-free survival compared to chemotherapy, particularly in early-stage patients. - SPG601 for Fragile X Syndrome: The FDA cleared SPG601 for a phase 2a trial in Fragile X Syndrome, addressing synaptic function through BK channel activation. This marks an advance for treating the core symptoms of the most common inherited intellectual disability. - Lumisight for Visualizing Breast Cancer: The FDA approved Lumisight and the Lumicell Direct Visualization System for use during lumpectomy surgeries to detect residual cancer tissues. This system, shown in the INSITE trial, improves surgical outcomes by reducing the need for second operations. VIsit Nascentmc.com for medical writing assistance. Visit learnamastyle.com for freebies on medical writing and editing and ChatGPT.

Visit nascentmc.com/podcast for full show notes. - The FDA has expanded the approval of benralizumab (Fasenra) to include children aged 6 to 11 with severe eosinophilic asthma, building on its previous approval for patients aged 12 and older. Benralizumab is a monoclonal antibody that targets and reduces eosinophilic inflammation, crucial in the severe asthma pathway. This approval was supported by the Phase 3 TATE study, which confirmed that the drug's effects in younger children were consistent with those seen in older patients. - The FDA has approved new administration methods for cenobamate (Xcopri), allowing the tablets to be crushed for oral suspension or delivered via nasogastric tube for adults with partial-onset seizures. Cenobamate, initially approved in 2019, works by inhibiting neuronal firing and modulating the GABA ion channel. The approval follows a study confirming that these new methods are bioequivalent to the original oral intake of intact tablets. - The FDA's advisory committee has recommended adopting minimal residual disease (MRD) as a new endpoint for the accelerated approval of multiple myeloma treatments. This recommendation is based on the EVIDENCE study, which highlighted MRD's ability to detect very low levels of disease, offering a quicker and more sensitive measure of treatment efficacy. If adopted, MRD could allow for faster approvals and potentially better patient outcomes in multiple myeloma.  - Labcorp's Mpox PCR Test Home Collection Kit has received FDA Emergency Use Authorization for diagnosing mpox in adults, marking the first at-home collection kit for this purpose. The kit uses PCR technology to detect the virus from lesion swabs collected by patients at home. This development comes in response to rising mpox cases and aims to improve accessible diagnostic options during public health emergencies. - The FDA has approved the use of dolutegravir/lamivudine (Dovato) for HIV treatment in adolescents, making it the first oral, two-drug, single-tablet regimen for this age group. The combination targets crucial steps in the HIV replication process and aims to simplify lifelong treatment regimens, enhancing adherence. Approval was based on the DANCE study's findings, which demonstrated effective viral suppression in adolescents, consistent with adult data. Vist LearnAMAStyle.com for several essential tip sheets that you can download as well as a free course on using AI in medical writing, and an AMA style quiz to test your knowledge

·       Nascentmc.com for medical writing assistance for your company.Visit nascentmc.com/podcast for full show notes Cilta-cel for Myeloma: The FDA approved ciltacabtagene autoleucel (Carvykti; cilta-cel) for adults with relapsed or refractory multiple myeloma who have tried at least one prior therapy including a proteasome inhibitor and an immunomodulatory agent, and are refractory to lenalidomide. This CAR T-cell therapy, initially approved in 2022, was confirmed effective in the phase 3 CARTITUDE-4 study, showing significant reduction in disease progression or death risk by 59% compared to standard care. Enhertu for HER2-positive Solid Tumors: Fam-trastuzumab deruxtecan-nxki (Enhertu) received FDA approval for treating unresectable or metastatic HER2-positive solid tumors in adults who have had previous systemic treatment and lack satisfactory alternative options. This therapy, a conjugate of an anti-HER2 antibody and a cytotoxic drug, was first approved in 2019 and targets HER2-expressing cancer cells to potentially minimize damage to normal tissues. Fanapt for Bipolar: Iloperidone (Fanapt) has been approved for the acute treatment of manic or mixed episodes in adults with bipolar I disorder. Previously approved for schizophrenia, iloperidone targets neurotransmitters like dopamine and serotonin. It demonstrated efficacy in a pivotal trial, showing significant improvement on the Young Mania Rating Scale. Zevtera for Multiple Bacterial Infections: Ceftobiprole medocaril sodium (Zevtera) was approved for treating adults with Staphylococcus aureus bloodstream infections, right-sided infective endocarditis, and acute bacterial skin and skin structure infections. Also approved for pediatric community-acquired bacterial pneumonia, ceftobiprole is a broad-spectrum cephalosporin that combats various bacteria including MRSA. TriClip for Tricuspid Regurgitation: The FDA approved the TriClip™ transcatheter edge-to-edge repair system for treating tricuspid regurgitation. This minimally invasive option clips the tricuspid valve leaflets to improve blood flow and prevent the need for surgery. The TRILUMINATE Pivotal trial showed significant improvements in TR severity and quality of life with a good safety profile. Revumenib for Acute Leukemia: The FDA granted priority review to revumenib (SNDX-5613) for treating adult and pediatric patients with relapsed or refractory acute leukemia with KMT2A rearrangements. As a new therapeutic agent, revumenib inhibits the menin-MLL protein interaction crucial in leukemic transformation. Early trial results show promising remission rates, with a PDUFA action date scheduled for September 26, 2024.  

Visit nascentmc.com/podcast for full show notes Winrevair for PAH: Sotatercept-csrk (Winrevair) is FDA-approved for treating adults with pulmonary arterial hypertension (PAH), enhancing exercise capacity, improving WHO functional class, and reducing clinical worsening events. It's the first FDA-approved activin signaling inhibitor for PAH, addressing the imbalance in vascular cell proliferation underlying the condition. The approval is based on the Phase 3 STELLAR trial, demonstrating significant improvements in walk distance and reduced risk of clinical worsening events [1]. Vafseo for CKD: Vadadustat (Vafseo) is approved for managing anemia due to chronic kidney disease (CKD) in adult dialysis patients. As an oral HIF-PH inhibitor, it stimulates endogenous erythropoietin production, offering a novel approach to anemia management. Approval is supported by efficacy and safety data from the INNO2VATE program and post-marketing safety data from Japan [2]. Ultomiris for NMOSD: Ravulizumab-cwvz (Ultomiris) is FDA-approved for treating neuromyelitis optica spectrum disorder (NMOSD) in patients with anti-AQP4 antibodies. It's a terminal complement C5 inhibitor administered once every two months, demonstrating efficacy in preventing relapses. Approval is based on the Phase 3 CHAMPION-NMOSD study, showing significant reductions in relapse risk compared to placebo [3]. Evolut FX+ for TAVR: The Evolut™ FX+ transcatheter aortic valve replacement (TAVR) system is FDA-approved for treating symptomatic severe aortic stenosis. It features enhancements for improved catheter maneuverability without compromising effectiveness. The approval expands treatment options for patients across all risk categories [4]. Vemlidy for Pediatric HBV: Tenofovir alafenamide (Vemlidy) is FDA-approved for treating chronic hepatitis B virus (HBV) infection in pediatric patients aged 6 years and older with compensated liver disease. It's a preferred or first-line treatment option, addressing a significant medical need. Approval is based on the Phase 2 clinical trial 1092, demonstrating efficacy and safety in this patient population [5]. Pemgarda for COVID: Pemgarda, a monoclonal antibody, is FDA-approved for preventive use in immunocompromised individuals aged 12 and older against COVID-19. It prevents virus attachment and has shown promising results in reducing symptomatic COVID-19 cases. Approval is based on emergency use authorization and preliminary data from the CANOPY Phase 3 clinical trial [6].

Nascentmc.com for medical writing assistance for your company. Visit nascentmc.com/podcast for full show notes Visit learnAMAstyle.com for free downloads regarding editing and the AMA Manual of Style tip sheet.  Nexletol and Nexlizet for LDL Lowering and CV Risk: The FDA approved bempedoic acid (Nexletol) and its combination with ezetimibe (Nexlizet) for reducing cardiovascular risk and treating primary hyperlipidemia. Bempedoic acid inhibits a cholesterol synthesis enzyme, while ezetimibe blocks cholesterol absorption, both lowering LDL-C levels. The approval, for high-risk patients not yet having cardiovascular events, was based on the CLEAR Outcomes trial. Iclusig for ALL: The FDA granted accelerated approval to ponatinib (Iclusig) with chemotherapy for treating previously untreated Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Ponatinib, a tyrosine kinase inhibitor, is the first targeted agent for this leukemia type in combination with chemotherapy, based on the PhALLCON trial results. Further confirmatory data may be required to verify its clinical benefit. Elahere for Ovarian Cancer: Mirvetuximab soravtansine-gynx (Elahere) received FDA approval for treating folate receptor alpha-positive, platinum-resistant ovarian cancer, marking it as the first antibody-drug conjugate in the U.S. for this condition. The treatment targets cancer cells by binding to the folate receptor alpha, delivering a cytotoxic agent. Approval was based on the Phase 3 MIRASOL trial. Duvyzat for DMD: The FDA approved givinostat (Duvyzat) for patients 6 years or older with Duchenne muscular dystrophy (DMD), a rare neuromuscular condition. Givinostat is an HDAC inhibitor that mitigates muscle damage and slows disease progression. Approval was based on the EPIDYS trial results, granted to Italfarmaco S.p.A. Spevigo for Psoriasis: Spesolimab-sbzo (Spevigo) received FDA approval for treating generalized pustular psoriasis (GPP) in adults and pediatric patients, expanding its indication from initial approval for GPP flares. As the first targeted therapy for GPP, it acts as an IL-36 receptor antagonist. The expanded approval was based on the Effisayil 2 trial, showing significant reduction in GPP flares. Tryvio for Hypertension: The FDA approved aprocitentan (Tryvio) in combination with other antihypertensive drugs for adults not adequately controlled on other medications, marking it as the first new oral antihypertensive therapy pathway in nearly 40 years. Based on the PRECISION trial, aprocitentan was shown to be effective in patients with resistant hypertension. Opsynvi for PAH: Macitentan and tadalafil (Opsynvi) was approved by the FDA for adults with pulmonary arterial hypertension (PAH) and WHO functional class II-III, as the first once-daily single-tablet combination therapy for PAH. The approval was based on the A DUE study, demonstrating greater reduction in pulmonary vascular resistance compared to monotherapies. Lenmeldy for Juvenile Metachromatic Leukodystrophy: The FDA approved Lenmeldy (atidarsagene autotemcel) gene therapy for children with metachromatic leukodystrophy (MLD), a rare genetic disease affecting the brain and nervous system. The therapy uses the patient's own genetically modified stem cells to produce the missing enzyme. Approval was based on significant improvements in survival, mobility, and cognitive functions observed in clinical trials and an expanded access program.

Please visit nascentmc.com/podcast for all the details.  Go to learnAMAstyle.com for lots of freebies on AMA Style and the use of AI in medical writing and editing Here is information on the latest US FDA approvals, the week of March 11 – March 15, 2024 Liso-cel for CLL/SLL   - The FDA approved lisocabtagene maraleucel (liso-cel; Breyanzi) for adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least two prior therapies. It is a CAR T-cell therapy that modifies patient's T cells to target tumor antigens. The approval was based on the TRANSCEND CLL 004 study, showing a 20% complete response rate and a median duration of response not reached by the data cutoff. Tislelizumab for Esophageal SCC   - Tislelizumab-jsgr (Tevimbra) received FDA approval for unresectable or metastatic esophageal squamous cell carcinoma (ESCC) patients after prior systemic chemotherapy. The approval was based on the phase 3 RATIONALE 302 trial, which showed significant improvement in overall survival compared to chemotherapy (8.6 months vs. 6.3 months). This marks a critical advancement for patients with limited treatment options after first-line failures. Resmetirom for NASH   - The FDA approved resmetirom (Rezdiffra) for adults with non-cirrhotic non-alcoholic steatohepatitis (NASH) with moderate to advanced fibrosis, to be used alongside diet and exercise. This is the first medication approved to directly address liver damage in NASH patients with significant liver scarring, acting as a partial activator of a thyroid hormone receptor to reduce liver fat. The approval, based on a 54-month trial, used a surrogate endpoint at 12 months to demonstrate improvement in liver scarring and inflammation. Maralixibat for Cholestatic Pruritus   - Maralixibat (Livmarli) oral solution was approved for treating cholestatic pruritus in patients aged 5 years and older with progressive familial intrahepatic cholestasis (PFIC). It is an orally administered ileal bile acid transporter inhibitor, showing efficacy in the Phase 3 MARCH clinical trial across various genetic types of PFIC. Additionally, a higher concentration formulation is under consideration to extend its use to younger PFIC patients. Guselkumab for UC   - A supplemental Biologics License Application (sBLA) has been submitted for guselkumab (Tremfya) for treating adults with moderate-to-severely active ulcerative colitis (UC). The submission is based on the QUASAR program results, demonstrating significant clinical remission at Week 44 compared to placebo. Guselkumab, a novel IL-23 inhibitor, has previously been approved for moderate-to-severe plaque psoriasis and active psoriatic arthritis, marking its potential expansion into UC treatment.  

Here is information on the latest US FDA approvals, the week of March 4 – March 8, 2024 ·         ChatGPT4 in medical writing and editing—visit learnAMAstyle.com ·       Nascentmc.com for medical writing assistance for your company. Visit nascentmc.com/podcast for full show notes   - **OTC Glucose Monitor**: The FDA has approved the Dexcom Stelo Glucose Biosensor System for over-the-counter sale, a first for a continuous glucose monitor. Designed for people aged 18 and older not using insulin, it helps manage diabetes with oral medications or monitors the impact of diet and exercise on blood sugar levels. Scheduled for release in Summer 2024, the system offers a 15-day sensor wear time and does not alert users to low blood sugar episodes.   - **Semaglutide in CVD Risk**: The FDA has approved semaglutide (Wegovy) for reducing cardiovascular risk in adults with known heart disease who are overweight or obese, specifically targeting the reduction of major adverse cardiovascular events. This approval makes semaglutide the first weight-loss medication also indicated for preventing life-threatening cardiovascular events in patients with established cardiovascular disease and obesity or overweight.   - **Juvederm Additional Indication**: JUVÉDERM® VOLUMA® XC, a hyaluronic acid dermal filler, has received FDA approval for treating moderate to severe temple hollowing in adults over 21, marking it as the first HA filler for this purpose. With effects lasting up to 13 months, clinical studies show significant improvement and patient satisfaction with facial symmetry post-treatment. This approval highlights Allergan Aesthetics' commitment to innovation in aesthetic treatments.   - **Nivolumab in mUC**: The FDA approved nivolumab in combination with cisplatin and gemcitabine for first-line treatment of metastatic urothelial carcinoma, based on significant improvements in survival outcomes from the CHECKMATE-901 trial. This expands nivolumab's indications, which include treatments for melanoma and lung cancer, among others, demonstrating its broad applicability in cancer treatment.   - **Zanubrutinib in FL**: The FDA has granted accelerated approval to zanubrutinib and obinutuzumab for relapsed or refractory follicular lymphoma patients after two or more systemic therapies. This combination targets key pathways in B cell survival, offering a new treatment option for patients. Approval was based on the ROSEWOOD trial, highlighting significant patient outcome improvements.   - **Donanemab and Alzheimer's**: The FDA has postponed the decision on the approval of Eli Lilly's donanemab for Alzheimer's treatment to convene an advisory meeting for further examination of safety and efficacy data, indicating a significant delay. This reflects the complex nature of Alzheimer's drug approval and Eli Lilly's confidence in donanemab's potential benefits.   - **Tocilizumab Biosimilar**: Tyenne® (tocilizumab-aazg), the first FDA-approved biosimilar to Actemra® for various inflammatory diseases, is now available in both IV and subcutaneous formulations. This approval introduces a new treatment option for patients with conditions like rheumatoid arthritis and juvenile idiopathic arthritis, emphasizing advancements in biosimilar medications.   - **Denosumab Biosimilars**: The FDA approved Jubbonti and Wyost as interchangeable biosimilars to Prolia and Xgeva, respectively, marking a first for biosimilars targeting the RANKL inhibitor used in osteoporosis and cancer-related bone conditions. These approvals offer new treatment options for managing bone health, underlining the importance of biosimilar development in expanding patient care.   - **Clobetasol Propionate Eye Drops**: The FDA's approval of clobetasol propionate 0.05% eye drops for post-operative eye inflammation and pain introduces the first ophthalmic formulation of this corticosteroid and the first new steroid in ophthalmology in over 15 years. Developed using proprietary nanoparticle technology for twice-daily dosing, this approval offers a new option for effective pain and inflammation management post-eye surgery.

Visit nascentmc.com/podcast for full show notes Visit learnamastyle.com for the free course on ChatGPT4 for medical writers and editors.  - The FDA has approved amivantamab (Rybrevant) in combination with chemotherapy (carboplatin-pemetrexed) for the first-line treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test. This approval converts the May 2021 accelerated approval of amivantamab to full approval. - The FDA approved the AGENT Drug-Coated Balloon (DCB) for treating in-stent restenosis in coronary artery disease patients, introducing the first drug-coated coronary balloon in the US. It uses a paclitaxel-coated catheter to deliver medication directly to the vessel wall, offering an alternative to traditional treatments and aiming to reduce recurrence risks. The approval was based on the AGENT IDE trial, demonstrating its effectiveness over uncoated balloon angioplasty in reducing lesion failure, with no significant adverse events reported. - The FDA approved an oral suspension form of ibrutinib (Imbruvica) for several conditions, including chronic lymphocytic leukemia and chronic graft versus host disease, addressing the needs of patients who have difficulty swallowing pills. This is the first oral suspension formulation of a Bruton's tyrosine kinase inhibitor, originally approved in 2013 for mantle cell lymphoma. The approval, granted to Johnson & Johnson and Pharmacyclics, aims to simplify treatment for patients challenged with pill-swallowing. - Epcoritamab-bysp (Epkinly) received FDA approval for treating adult patients with relapsed or refractory follicular lymphoma after at least two prior therapies, marking it as the first subcutaneously administered bispecific antibody for this condition. It works by targeting both CD3 on T cells and CD20 on B cells to induce cell death. Based on the EPCORE NHL-1 trial results, this approval extends its use beyond diffuse large B-cell lymphoma, with AbbVie and Genmab sharing commercial responsibilities.  - The FDA granted priority review to the New Drug Application for govorestat (AT-007), a treatment for classic galactosemia, potentially the first of its kind if approved. Govorestat, an aldose reductase inhibitor, aims to reduce harmful galactitol levels, based on phase 3 study results among pediatric patients. Applied Therapeutics announced a PDUFA target action date of August 28, 2024, highlighting the urgent need for treatments in this area. - Obeticholic acid (Ocaliva) received FDA consideration for an expanded application to treat primary biliary cholangitis, building on its 2016 accelerated approval. It's designed for patients with or without cirrhosis, showing promise in post-marketing studies to confirm its clinical benefits. The review includes data from various studies and real-world evidence, with a PDUFA target date of October 15, 2024, aiming to address the needs of this autoimmune liver disease population.  - The FDA approved Biktarvy for HIV patients with suppressed viral loads who exhibit M184V/I resistance, offering a new treatment option for those with specific resistance mutations. Biktarvy combines three therapies in a single tablet, based on successful 48-week study data. Manufactured by Gilead Sciences, this approval expands treatment possibilities for patients facing resistance challenges. - The FDA rejected Minerva Neurosciences' New Drug Application for roluperidone as a treatment for schizophrenia's negative symptoms, citing insufficient evidence from a single study and lack of comprehensive data. Despite showing promise in targeting specific brain receptors, the FDA's concerns highlight the need for more extensive research and data to confirm its effectiveness and safety. Minerva plans to engage with the FDA to address these issues.

·       ChatGPT4 in medical writing and editing FREE COURSE at learnAMAstyle.com ·       Nascentmc.com for medical writing assistance for your company. Visit nascentmc.com/podcast for full show notes  Enmetazobactam for cUTI: The FDA approved enmetazobactam (Exblifep) for complicated urinary tract infections (cUTIs) in adults, offering a new weapon against antimicrobial-resistant gram-negative bacteria. It's combined with cefepime, based on successful phase 3 trial results, and is expected to hit the US market soon, addressing the needs of approximately 3.6 million US patients annually. Orchid Pharma receives approval, highlighting the drug's priority review status due to its Qualified Infectious Disease Product designation. Dupilumab for COPD: Dupilumab (Dupixent) is under priority review by the FDA as a novel biologic therapy for certain adults with uncontrolled chronic obstructive pulmonary disease (COPD), based on promising phase 3 trials. It targets interleukin signaling involved in COPD's inflammatory process, potentially reducing exacerbations and improving lung function over time. If approved, it would be a significant advancement for COPD treatment, offered by Regeneron Pharmaceuticals and Sanofi.  Melphalan Flufenamide Withdrawal: The FDA has withdrawn approval for melphalan flufenamide (Pepaxto) for certain multiple myeloma patients due to a lack of confirmed clinical benefit and safety concerns, marking the first application of new procedures for accelerated approval withdrawal. The decision follows the Food and Drug Omnibus Report Act amendments, concluding a process allowing manufacturer Oncopeptides an appeal opportunity. Teclistamab Biweekly Dosing: Teclistamab-cqyv (Tecvayli) receives FDA approval for biweekly dosing in relapsed or refractory multiple myeloma patients who have maintained a complete response for six months, enhancing treatment flexibility. This approval is based on the phase I/II MajesTEC-1 study, demonstrating maintained responses and safety with the new dosing schedule, highlighting fewer severe infections and no new safety concerns.  Linvoseltamab in RRMM: The FDA has granted priority review to the biologics license application for linvoseltamab for treating adult patients with relapsed or refractory multiple myeloma after at least three prior therapies. Targeted for an August 2024 decision, linvoseltamab aims to enhance T-cell activation against cancer cells, supported by promising results from the ongoing LINKER-MM1 trial, offering a new therapeutic option from Regeneron. ·       ChatGPT4 in medical writing and editing at learnAMAstyle.com ·       Nascentmc.com for medical writing assistance for your company. Visit nascentmc.com/podcast for full show notes

`    Free course on ChatGPT4 in medical writing and editing at learnAMAstyle.com ·       Nascentmc.com for medical writing assistance for your company. Visit nascentmc.com/podcast for full show notes Lifileucel for Melanoma: Lifileucel (Amtagvi) has received accelerated FDA approval for unresectable metastatic melanoma in adults previously treated with specific inhibitors, marking it as the first FDA-approved T cell therapy for solid tumor cancer. The therapy involves collecting and expanding a patient's own T cells from their tumor for reinfusion, demonstrating a 31.5% objective response rate in a global study. A phase 3 trial is in progress to confirm its clinical benefit. Iloprost Injection for Frostbite: The FDA approved Aurlumyn (iloprost) injection, the first-ever treatment for severe frostbite in adults to reduce amputation risk. Iloprost, a vasodilator, showed effectiveness in an open-label trial of 47 adults, offering a significant advancement in managing severe frostbite. MDMA-Assisted Therapy for PTSD: The FDA is considering a new drug application for MDMA-assisted psychotherapy for PTSD, a potential first federal approval of its kind. Supported by two Phase 3 clinical trials showing safety and efficacy, this therapy represents a novel treatment avenue for severe PTSD. NALIRIFOX for Pancreatic Cancer: NALIRIFOX, a combination chemotherapy regimen, has been approved as a first-line treatment for metastatic pancreatic cancer, the first such approval in over a decade. Demonstrating a survival benefit in phase 3 trials, NALIRIFOX offers a new standard for previously untreated patients. Tepotinib for NSCLC: The FDA has fully approved tepotinib (Tepmetko) for NSCLC with MET exon 14 skipping alterations, following accelerated approval in 2021. The approval is based on significant response rates and median overall survival benefits shown in the VISION trial. Omalizumab for Food Allergies: Omalizumab (Xolair) injection is the first FDA-approved medication to reduce the severity of allergic reactions to multiple foods from accidental exposure. Approved for patients aged 1 year and older, it's based on phase 3 trial results showing significant efficacy in preventing reactions to common allergens. Budesonide Oral Suspension for EOE: The FDA approved budesonide oral suspension (Eohilia) as the only oral therapy for eosinophilic esophagitis in patients 11 years and older. This approval offers a specific treatment option for EoE, a chronic disease causing esophageal inflammation and related symptoms. TriClip® for Tricuspid Regurgitation: The FDA approved the TriClip™ TEER system by Abbott for minimally invasive treatment of tricuspid regurgitation, a first-of-its-kind device. Based on the TRILUMINATE trial, the TriClip showed significant improvements in patients' condition and quality of life. ACE2016 for Solid Tumors: The FDA cleared the IND application for ACE2016, an allogeneic gamma delta 2 T cell therapy targeting EGFR-expressing solid tumors. Set to begin in 2024, this Phase 1 trial will evaluate ACE2016's safety, tolerability, and pharmacodynamics. Human Acellular Vessel (HAV) for Vascular Trauma: The FDA granted Priority Review for Humacyte's BLA for the HAV for urgent arterial repair in vascular trauma cases. Supported by clinical trial and real-world evidence, the HAV offers a bioengineered alternative for arterial repair without the need for immune suppression.           Free course on ChatGPT4 in medical writing and editing at learnAMAstyle.com ·       Nascentmc.com for medical writing assistance for your company. Visit nascentmc.com/podcast for full show notes  

Visit nascentmc.com/podcast for full show notes [free course] ChatGPT4 in medical writing and editing at learnAMAstyle.com Nascentmc.com for medical writing assistance for your CME or Medical Communications company. Eplontersen for ATTR-CM Eplontersen received FDA Fast Track designation for treating transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults, aiming to inhibit TTR protein production. Nivolumab for NSCLC The FDA accepted supplemental applications for nivolumab in resectable stage 2A to 3B non-small cell lung cancer (NSCLC) based on the CheckMate-77T trial showing improved survival rates. RSV Vaccine for Adults Aged 50 to 59 The FDA prioritizes review of GSK's Arexvy vaccine for RSV in adults aged 50-59 at risk of complications, expanding from its existing approval for those 60 and older. Spinal Cord Stimulation System The FDA approved Boston Scientific's WaveWriter Spinal Cord Stimulation Systems for chronic low back and leg pain treatment in non-surgery patients, based on the SOLIS trial results. OK-101 for Neuropathic Corneal Pain The FDA approved an IND application for OK-101, a first for treating neuropathic corneal pain (NCP), an Orphan disease, developed by OKYO Pharma Limited. Viz ICH Plus for Brain Bleed The FDA cleared Viz ICH Plus, an AI algorithm by Viz.ai for automating the identification and quantification of brain bleeds and structures in NCCT images. Vepdegestrant for MBC Vepdegestrant received FDA Fast Track designation for treating ER-positive/HER2-negative advanced or metastatic breast cancer in patients previously treated with endocrine therapy. VerTouch Spinal Puncture Device The FDA cleared VerTouch, a handheld imaging tool by IntuiTap Medical, designed to improve the accuracy of spinal punctures by providing a 2D image of lumbar spinal anatomy.

ChatGPT4 in medical writing and editing at learnAMAstyle.com Nascentmc.com for medical writing assistance for your company. Visit nascentmc.com/podcast for full show notes Tricuspid Valve Replacement System for Tricuspid Regurgitation The FDA approved the Evoque tricuspid valve replacement system, a first in the U.S. for a transcatheter tricuspid device, after the TRISCEND II trial showed significant improvements in TR grade and patient symptoms. TR, where the heart's valve does not close properly causing blood backflow, can now be treated with this device, which also received CE Mark approval in Europe and is produced by Edwards Lifesciences. Afami-Cel for Synovial Sarcoma The FDA is prioritizing the review of afamitresgene autoleucel (afami-cel) for advanced synovial sarcoma, based on positive results from the SPEARHEAD-1 trial showing a 39% response rate and increased survival rates. Afami-cel targets MAGE-A4 in synovial sarcoma, a rare soft tissue sarcoma, offering a new treatment option for this aggressive disease. It's manufactured by Adaptimmune Therapeutics with a decision expected by August 4, 2024.  Pulsed Field Ablation for Atrial Fibrillation Boston Scientific's FARAPULSE PFA System has been FDA approved for treating intermittent atrial fibrillation, offering a non-thermal, tissue-selective ablation alternative with proven safety and efficacy. The approval was based on the ADVENT study and real-world data, highlighting shorter ablation times and no severe side effects. Boston Scientific plans an immediate U.S. launch. Shorter Turnaround Time for Axi-cel The FDA approved a manufacturing process change for axi-cel (Yescarta), reducing delivery time from 16 to 14 days, which is a CD19-directed CAR T-cell therapy for certain lymphomas. This change, granted to Kite, a Gilead Sciences subsidiary, aims to improve treatment accessibility by offering faster delivery of this personalized therapy. AI Algorithm for Cervical Cancer Screening Hologic's Genius™ Digital Diagnostics System with the Genius™ Cervical AI algorithm has been FDA approved, introducing the first digital cytology platform integrating AI for cervical cancer screening. This system digitizes traditional Pap test slides, applying AI to enhance detection of pre-cancerous and cancerous cells, improving sensitivity and enabling remote case review. It will be available in the U.S. in early 2024. Trastuzumab Deruxtecan for Solid Tumors The FDA granted priority review to trastuzumab deruxtecan for treating unresectable or metastatic HER2-positive solid tumors, potentially marking it as the first HER2-directed, tumor-agnostic therapy. Based on the DESTINY-PanTumor02 study, showing promising survival outcomes, a decision is expected in the second quarter of 2024. The drug is developed by AstraZeneca and Daiichi Sankyo.  

Free course ChatGPT4 in medical writing and editing at learnAMAstyle.com ·       Nascentmc.com for medical writing assistance for your company.Visit nascentmc.com/podcast for full show notes Boxed Warning for CAR-T Cell Therapy: The FDA mandates a boxed warning on all CAR T-cell therapies due to increased secondary cancer risks, affecting six named treatments. This follows investigations into T-cell malignancies in patients treated with BCMA- or CD19-directed therapies. Manufacturers have 30 days to comply or challenge this requirement, reflecting the FDA's stance that benefits still outweigh risks.  Dupilumab for Pediatric EOE: The FDA has approved dupilumab for children aged 1-11 with eosinophilic esophagitis (EoE), expanding from its prior approval for older patients. Dupilumab, targeting interleukin-4 and -13, treats EoE, an allergic inflammation causing symptoms like heartburn and difficulty swallowing. The approval, based on the EoE KIDS trial, marks the first FDA-approved EoE treatment for this age group.  Wearable Device for Osteopenia: The FDA has cleared Osteoboost, a wearable belt for treating osteopenia, marking a non-drug medical device approach in this field. Developed by Bone Health Technologies, it uses low-intensity vibrations on the spine and hip, inspired by NASA's bone loss prevention methods. Clinical trials show its effectiveness in maintaining bone health, and it was reviewed under the FDA's De Novo process with Breakthrough Device Designation.  BSI-082 IND for Solid and Liquid Tumors: The FDA approved an IND application for BSI-082, a novel antibody targeting SIRPα and β to enhance tumor-associated macrophages' phagocytic activity. Developed by Biosion USA, Inc., BSI-082 covers over 90% of human populations and avoids broad toxicity typical in CD47-targeting therapies. Its in vivo efficacy, especially when combined with other antibodies, shows significant anti-tumor effects in animal models. VCA-894A for Charcot Marie-Tooth: The FDA approved an IND application for VCA-894A by Vanda Pharmaceuticals to treat Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S). VCA-894A, an antisense oligonucleotide, targets the IGHMBP2 gene variant responsible for CMT2S. CMT2S, a rare condition with symptoms like muscle weakness and sensory impairment, currently has no available treatments; this approval represents a significant step in addressing this genetic disorder. Free course on ChatGPT4 for medical writers and editors learnAMAstyle.com

For the free course "ChatGPT4 for Medical Writers and Editors," go to learnAMAstyle.com Visit Nascentmc.com/podcast for detailed show notes and links.         Erdafitinib for Urothelial Carcinoma: The FDA has fully approved erdafitinib (Balversa) for advanced or metastatic urothelial carcinoma with FGFR3 genetic alterations in adults. This follows its initial accelerated approval and is based on the phase 3 THOR trial results, showing improved survival rates and manageable side effects. Erdafitinib reduced death risk by 36% and had a lower treatment discontinuation rate compared to chemotherapy.        TTFields in NSCLC: The FDA is reviewing a premarket approval application for tumor treating fields (TTFields) combined with standard therapies for platinum-resistant non–small cell lung cancer. TTFields, first approved in 2011, disrupt cancer cell division and showed enhanced survival in NSCLC patients when combined with immune checkpoint inhibitors or docetaxel, without increasing systemic toxicities. The FDA's decision is expected in the second half of 2024.        AI-Powered Device to Detect Skin Cancer: The FDA has approved the first AI-powered handheld device by DermaSensor for assisting in skin cancer detection. It uses AI-driven spectroscopy for analyzing skin lesions and is based on a study involving over 1000 patients. While not a primary screening tool, it aids in detecting melanoma and other skin cancers, especially in patients over 40, and requires further validation testing.        Casgevy for Transfusion-Dependent Beta Thalassemia: The FDA has approved Casgevy (exa-cel), developed by Vertex Pharmaceuticals and CRISPR Therapeutics, for treating transfusion-dependent beta-thalassemia. This follows its approval for sickle cell disease and marks the first CRISPR gene-editing technology application for this condition. The approval came ahead of the anticipated date and follows Bluebird Bio's 2022 approval for a similar gene therapy.        HyQvia for CIDP: HyQvia, an immune globulin infusion 10%, has been approved by the FDA for chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. Initially approved for primary immunodeficiency, HyQvia is the only product combining immunoglobulin with hyaluronidase, allowing for monthly subcutaneous infusions. The approval is based on its demonstrated efficacy in preventing neuromuscular disability relapse.        Physicians' Understanding of FDA Approval Process: A national survey reveals that many physicians have limited understanding of the FDA's drug and medical device approval processes. Only 41% of the surveyed physicians reported moderate or better comprehension of the drug approval process. Despite believing in the adequacy of FDA standards, there's a call for more rigorous post-marketing studies and enhanced education on FDA processes to avoid misconceptions and inaccurate patient advice.

Visit nascentmc.com to get medical writing assistance for your company. Visit learnAMAstyle.com for free downloads about medical editing and writing in the AI era. See full write up and supporting links for this episode at nascentmc.com/podcast. Summary: §  FDA has approved berdazimer topical gel, 10.3% (Zelsuvmi), as the first novel drug for molluscum contagiosum, a viral skin infection affecting adults and children over 1 year old.  §  Pembrolizumab (Keytruda) is now approved for stage III-IVA cervical cancer treatment in combination with chemoradiotherapy, based on positive data from the KEYNOTE-A18 trial. It's the third indication for cervical cancer and 39th overall  §  The FDA issued a drug safety communication regarding GLP-1 receptor agonists and suicidal thoughts, finding no clear relationship between them.  §  BrainSee, a noninvasive test, approved for predicting progression from amnestic mild cognitive impairment (aMCI) to Alzheimer's dementia using MRI and cognitive assessments, offering a same-day alternative to traditional methods. §  SH-105, a novel treatment for breast and ovarian cancers, had its NDA accepted with a target action date set for June 29, 2024, offering a differentiated injectable product. §  Medtronic's Percept RC deep brain stimulation system received FDA approval for Parkinson's. §  NexoBrid, a topically administered biological product for burn treatment, expanded its label to include pediatric patients based on positive results from clinical trials.  §  The FDA approved the Teneo excimer laser platform for LASIK surgery, featuring high-speed eye tracking and fast ablation times for precise vision correction in individuals aged 22 and older. See full write up and supporting links at nascentmc.com/podcast.

Use Your Scientific or Medical Background in Medical Editing & Factchecking so you can supplement your income while working from anywhere on your own schedule. Visit learnAMAstyle.com for a free step-by-step checklist. Get immediate medical writing assistance from experienced, excellent, native-English speaking MD- and PhD-medical writers. Nascentmc.com Today's episode: FDA approves first state request to import drugs from Canada: The FDA has approved Florida's request to import certain drugs from Canada, marking the first time a state has been authorized to buy lower-cost medications in bulk from abroad.https://www.cnn.com/2024/01/05/politics/florida-canada-drug-imports-fda/index.html https://www.flgov.com/2024/01/05/florida-becomes-first-in-the-nation-to-have-canadian-drug-importation-program-approved-by-fda/ ·       5 FDA decisions to watch for: o   Donanemab from Eli Lilly, if approved, could be a significant Alzheimer's treatment, with Phase 3 trial results showing substantial cognitive and functional decline slowing. o   Resmetirom, an oral thyroid hormone receptor agonist, manufactured by Madrigal Pharmaceuticals showed promise in addressing nonalcoholic steatohepatitis (NASH), a leading cause of liver transplantation. o   Sotatercept, acquired by Merck, is being evaluated for treating pulmonary arterial hypertension (PAH) based on promising results from the Phase 3 STELLAR trial. o   Lifileucel, in a phase 3 trial for melanoma, is the first tumor-infiltrating lymphocyte (TIL) therapy for advanced melanoma. FDA's decision on lifileucel is expected by mid-February, and it offers potential as a one-time cell therapy for solid tumor cancer. o   Odronextamab, developed by Regeneron Pharmaceuticals, could become the first bispecific antibody approved for both FL and DLBCL, common non-Hodgkin lymphoma subtypes. This content was independently written by Dr. Emma Hitt Nichols, but the idea for the 5 upcoming drugs came from this article https://www.biopharmadive.com/news/5-fda-approval-decisions-watch-first-quarter-2024/703377 Use Your Scientific or Medical Background in Medical Editing & Factchecking so you can supplement your income while working from anywhere on your own schedule. Visit learnAMAstyle.com for a free step-by-step checklist. Get immediate medical writing assistance from experienced, excellent, native-English speaking MD- and PhD-medical writers. Nascentmc.com 

Summary: Visit learnAMAstyle.com for the downloadable AMA Style cheat sheet Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast A summary of Eleven Potentially Practice-Changing Drugs Approved by the FDA in 2023 https://www.pharmacytimes.com/view/eleven-potentially-practice-changing-drugs-were-approved-by-the-fda-in-2023 Also added Casgevy and Lyfgenia for Sickle Cell Disease Podcast episode was from December 11, 2023. Happy new year!   Intro and outro music Garden Of Love by Pk jazz Collective

For the AMA Style cheat sheet learnamastyle.com See the full write ups for today's episode at nascentmc.com/podcast Here are the highlights:  Test for Risk of Opioid Addiction:The FDA has approved AvertD by SOLVD Health, a test for assessing opioid addiction risk in individuals over 18, using genetic variants to evaluate addiction risk before prescribing oral opioids for acute pain; however, it's met with skepticism and not intended for chronic pain patients. https://www.cnn.com/2023/12/20/health/opioid-use-disorder-test-avertd/index.html https://avertdtest.com/  Filsuvez for Junctional Epidermolysis Bullosa:Filsuvez, a birch triterpenes topical gel, received FDA approval for treating Junctional and Dystrophic Epidermolysis Bullosa (JEB/DEB) in individuals aged 6 months and older, marking the first FDA-approved therapy for JEB's partial thickness wounds and a significant advancement in managing this debilitating skin disorder.https://resources.chiesiusa.com/Filsuvez/FILSUVEZ_PI.pdf Budesonide for IgA Nephropathy:Budesonide (Tarpeyo) received full FDA approval for reducing kidney function loss in adults with IgA nephropathy (IgAN), targeting specific antibodies implicated in IgAN based on phase 3 NefIgArd clinical trial data, marking the first FDA approval for IgAN treatment based on kidney function measures. https://www.tarpeyo.com/prescribinginformation.pdf Eplontersen for hATTR Polyneuropathy:Eplontersen (Wainua) was approved by the FDA for treating polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis (hATTR-PN), based on the NEURO-TTRansform Phase III trial, and is the only hATTR-PN treatment that can be self-administered via an auto-injector. https://pubmed.ncbi.nlm.nih.gov/37768671/ https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/217388s000lbl.pdf NRX-101 for Complicated UTI:The FDA cleared an Investigational New Drug application for NRX-101, a D-cycloserine and lurasidone blend developed by NRx Pharmaceuticals for treating complicated urinary tract infections, with a pending request for Qualified Infectious Disease Product designation. https://www.prnewswire.com/news-releases/nrx-pharmaceuticals-announces-fda-clearance-of-its-investigational-new-drug-ind-application-for-nrx-101-in-the-treatment-of-complicated-urinary-tract-infections-302017523.html?utm_source=pocket_saves Gefapixant for Chronic Cough:Merck's gefapixant, a selective antagonist of P2X3 receptors for treating chronic cough, was rejected by the FDA for the second time due to insufficient evidence of effectiveness in treating refractory or unexplained chronic cough in adults. https://www.businesswire.com/news/home/20231220302305/en/Merck-Provides-U.S.-Regulatory-Update-on-Gefapixant V116 for Invasive Pneumococcal Disease:Merck's V116, a 21-valent pneumococcal conjugate vaccine, was accepted by the FDA for priority review to prevent invasive pneumococcal disease in adults, based on STRIDE-3 Phase 3 trial data, with a target action date set for June 17, 2024. https://www.merck.com/news/fda-grants-priority-review-to-mercks-new-biologics-license-application-for-v116-an-investigational-21-valent-pneumococcal-conjugate-vaccine-specifically-designed-to-protect-adults Sotorasib in NSCLC:Lumakras (sotorasib), already under accelerated approval for KRASG12C-mutated non-small cell lung cancer (NSCLC), faces uncertainty for full FDA approval after an advisory committee voted against it, though it will remain available for clinical use.https://www.onclive.com/view/codebreak-200-sotorasib-in-second-line-kras-g12c-mutated-nsclc nascentmc.comlearnAMAstyle.com

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcastHere are the highlights: Belzutifan (Welireg) for RCC: - FDA approved belzutifan (Welireg) for advanced renal cell carcinoma (RCC) following prior treatments with PD-1/PD-L1 inhibitors and VEGF-TKI. Belzutifan is an HIF-2α inhibitor, the first novel therapeutic class for advanced RCC since 2015. Approval based on phase 3 LITESPARK-005 study data, granted to Merck Inc. Pulse Field Ablation (PFA) system for AFib: - FDA approved PulseSelect PFA system for atrial fibrillation (Afib) treatment. PFA uses electrical pulses to ablate cardiac tissue without thermal energy, reducing adverse events. Medtronic Inc. received approval. Roflumilast 0.3% (Zoryve) for Seborrheic Dermatitis: - FDA approved roflumilast foam 0.3% (Zoryve) for seborrheic dermatitis in individuals aged 9 and older. This is the first drug with a new mechanism of action for seborrheic dermatitis in over two decades. Approval supported by positive results from the STRATUM phase 3 trial, granted to Arcutis Biotherapeutics, Inc. Eflornithine (Iwilfin) for Neuroblastoma: - FDA approved eflornithine (Iwilfin) to reduce the risk of relapse in high-risk neuroblastoma patients. Eflornithine inhibits ornithine decarboxylase, reducing cell growth. Approval based on multi-site study results and granted to US WorldMeds, LLC. Isavuconazonium Sulfate For Pediatric Patients: - FDA approved isavuconazonium sulfate (Cresemba) for invasive aspergillosis and mucormycosis in pediatric patients. Provides an alternative to eye drops for glaucoma treatment, offering continuous delivery of travoprost. Approval based on Phase 3 trials and plans for label change, granted to Glaukos. Augmented Reality Smart Glasses Technology: - FDA approved NuLoupes augmented reality smart glasses with 3D stereoscopic imaging for dentistry and medicine. Offers live 3D stereoscopic imaging and plans to ship developer kits in 2024. Approval granted to NuEyes. OneRF Ablation System for Parkinson's: - FDA cleared the OneRF Ablation System for neurosurgical procedures in Parkinson's disease and other neurological conditions. Designed to capture electrical activity and selectively destroy brain tissue. Launch planned by NeuroOne in the first half of 2024. Pimicotinib For Tenosynovial Giant Cell Tumor: - FDA granted fast track approval to pimicotinib for the treatment of tenosynovial giant cell tumors. Demonstrated an overall response rate of 87.5% in a Phase 1b trial. Manufactured by Abbisko and commercialized in partnership with Merck. NDA for MDMA-Assisted Therapy for PTSD: - NDA submitted for MDMA in combination with psychotherapy for PTSD, aiming to be the first psychedelic-assisted therapy approved for PTSD. Submission by MAPS Public Benefit Corporation based on positive Phase 3 clinical trial results. Requested Priority Review of the NDA.

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast Here are the highlights: 1. Iptacopan (Fabhalta) for PNH: The FDA approved iptacopan (Fabhalta), the first oral monotherapy for adults with paroxysmal nocturnal hemoglobinuria (PNH), a condition characterized by the destruction of red blood cells. This approval, based on the APPLY-PNH and APPOINT-PNH studies, marks a significant advancement over existing infusion-based treatments, with most patients experiencing increased hemoglobin levels without the need for blood transfusions. 2. Casgevy and Lyfgenia for Sickle Cell Disease: The FDA approved two gene therapies, Casgevy and Lyfgenia, for sickle cell disease treatment. Casgevy, using CRISPR/Cas9 technology for a one-time stem cell transplant, showed significant effectiveness in reducing severe vaso-occlusive crises in trials. Lyfgenia, employing lentiviral vector gene addition, demonstrated a high rate of resolution in severe vaso-occlusive events, marking a substantial progression in treating this condition. 3. SGT-003 for Duchenne Muscular Dystrophy: SGT-003, a gene therapy for Duchenne muscular dystrophy (DMD), received FDA fast track designation. Utilizing a novel capsid to deliver microdystrophin, this one-time intravenous therapy aims to address the underlying cause of DMD. It follows the accelerated approval of another gene therapy, Elevidys, also for DMD, highlighting advancements in the treatment of this muscular disorder. 4. New Fingerstick Blood Collection Device: The FDA has cleared a new fingerstick blood collection device, MiniDraw™, by Becton Dickinson. This less invasive method for obtaining blood samples aims to transform diagnostic testing by allowing lab-quality results for common blood tests without the need for traditional venous blood draws, offering a more patient-friendly alternative.   See the full write ups for today's episode at nascentmc.com/podcast  Intro and outro music Garden Of Love by Pk jazz Collective      

Summary: Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast Here are the highlights: Nirogacestat (Ogsiveo) For Desmoid Tumors: Nirogacestat is the first FDA-approved drug for treating progressing desmoid tumors in adults, offering a significant improvement in response rate compared to placebo in a major trial. Pirtobrutinib (Jaypirca) in CLL/SLL: The FDA granted accelerated approval for pirtobrutinib to treat adult patients with CLL/SLL who have undergone at least two prior therapies, with its effectiveness based on response rates from the BRUIN trial. Enfortumab Vedotin (Padcev) With Pembrolizumab (Keytruda) for Urothelial Cancer: The FDA is reviewing a combination treatment of enfortumab vedotin and pembrolizumab for urothelial cancer under priority review, showing significant improvement in survival rates compared to chemotherapy in a Phase 3 study. Lisocabtagene Maraleucel (Breyanzi) for CLL/SLL: The FDA is reviewing lisocabtagene maraleucel for expanded use in treating CLL/SLL patients who have relapsed after BTKi and BCL2i treatments, based on positive results from the TRANSCEND CLL 004 study. Xanomeline-trospium (KarXT) for Schizophrenia: The FDA is considering xanomeline-trospium for treating schizophrenia, with potential benefits over traditional treatments in reducing common antipsychotic side effects, based on the EMERGENT program results. Roflumilast Cream for Atopic Dermatitis: The FDA is reviewing roflumilast cream for treating atopic dermatitis in adults and children, supported by positive results from the INTEGUMENT-1 and INTEGUMENT-2 studies, with a potential for simplification in disease control. See the full write ups for today's episode at nascentmc.com/podcast Intro and outro music Garden Of Love by Pk jazz Collective

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast   Here are the highlights: ·       Capivasertib (Truqap) has been FDA-approved for the treatment of hormone receptor-positive/HER2-negative breast cancer with AKT pathway alterations, showing improved progression-free survival in clinical trials. ·       Repotrectinib (Augtyro) has received FDA approval for ROS1-positive non-small cell lung cancer (NSCLC), demonstrating high response rates in patients who had not received a prior tyrosine kinase inhibitor. ·       Enzalutamide (Xtandi) has gained FDA approval for nonmetastatic castration-sensitive prostate cancer (nmCSPC) in high-risk patients with biochemical recurrence, expanding its use in prostate cancer treatment. ·       Pembrolizumab (Keytruda) has received supplemental approval for the first-line treatment of HER2-negative stomach cancer when combined with chemotherapy, showing improved overall survival compared to chemotherapy alone. ·       The Symplicity Spyral™ renal denervation (RDN) system has been approved by the FDA for treating hypertension through a minimally invasive procedure targeting overactive nerves near the kidneys.  ·       A combination of taurolidine and heparin catheter lock solution (DefenCath) has been approved for preventing catheter-related bloodstream infections in hemodialysis patients, demonstrating a significant reduction in infection risk in clinical trials.     Intro and outro music Garden Of Love by Pk jazz Collective

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at  nascentmc.com/podcast Here are the highlights: The FDA has approved Zepbound (tirzepatide) for adults with obesity, allowing its use in individuals with a body mass index (BMI) of 30 or more, as well as overweight individuals (BMI of 27 or greater) with at least one weight-related condition. Tirzepatide, an agonist of GLP-1 and GIP, was shown to significantly reduce body weight when used in combination with a reduced-calorie diet and increased physical activity in clinical trials, but it may have adverse effects, including gastrointestinal issues and a potential link to medullary thyroid cancer. Ixchiq has become the first FDA-approved chikungunya vaccine for individuals aged 18 and older at increased risk of exposure to the chikungunya virus, which is primarily transmitted by mosquitoes. Chikungunya is a global health threat with symptoms including fever, joint pain, and rash, and severe joint pain that can last for months. The vaccine, while effective, may cause severe or prolonged chikungunya-like adverse reactions, and the manufacturer, Valneva, is required to conduct a postmarketing study to assess these risks. Fruzaqla (fruquintinib) has received FDA approval for patients with metastatic colorectal cancer (mCRC) who have undergone previous treatments, including various chemotherapy and targeted therapies. Fruquintinib, a kinase inhibitor of VEGFRs, demonstrated improvements in overall survival and progression-free survival in clinical trials, with consistent safety profiles. The approval was granted to Takeda, and the drug is also under review for marketing authorization in Europe. Adzynma (ADAMTS13, recombinant-krhn) has been approved by the FDA for congenital thrombotic thrombocytopenic purpura (cTTP), making it the first FDA-approved recombinant ADAMTS13 protein for this rare condition. cTTP results from a deficiency in ADAMTS13, leading to blood clot formation and severe bleeding episodes. Adzynma serves as prophylactic and on-demand treatment, with promising results in a phase 3 study, and its approval was granted to Takeda. Intro and outro music Garden Of Love by Pk jazz Collective

Summary: Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast   Here are the highlights: Pembrolizumab (Keytruda) has received FDA approval for the treatment of metastatic biliary tract cancer, both in combination with chemotherapy and as a monotherapy. The approval is based on the positive outcomes of the KEYNOTE-966 trial, where patients receiving pembrolizumab plus chemotherapy demonstrated a statistically significant improvement in overall survival compared to those receiving a placebo with chemotherapy. Common adverse reactions included hematologic abnormalities, pyrexia, fatigue, cholangitis, and hepatic enzyme elevations. Approval was granted to Merck. Secukinumab (Cosentyx) has gained FDA approval for the treatment of moderate-to-severe hidradenitis suppurativa (HS), making it the first FDA-approved IL-17A inhibitor for this condition. Approval is based on results from the SUNSHINE and SUNRISE trials, showing significant improvements in response rates in HS patients treated with secukinumab compared to placebo. Ustekinumab-auub (Wezlana) has been granted FDA approval as an interchangeable biosimilar for Stelara, offering treatment options for multiple inflammatory diseases. It is indicated for moderate to severe plaque psoriasis, psoriatic arthritis, Crohn's disease, and ulcerative colitis in adult patients, as well as pediatric patients with plaque psoriasis and psoriatic arthritis. Vonoprazan (Voquezna) has received FDA approval for the treatment of erosive esophagitis (GERD). As a potent potassium-competitive acid blocker (PCAB), it offers an alternative to proton pump inhibitors (PPIs). Approval is based on the PHALCON-EE study, where vonoprazan demonstrated noninferiority to lansoprazole in healing GERD. Abatacept (Orencia) has been expanded for use in pediatric patients aged 2 years and older to treat psoriatic arthritis. Originally approved for rheumatoid arthritis in adults in 2005, abatacept was also approved for adult psoriatic arthritis in 2017. Exa-cel, a CRISPR-based therapy developed by CRISPR Therapeutics and Vertex, is under FDA review for sickle cell disease. The advisory panel has found it safe for clinical use, with potential approval expected in December. Exa-cel aims to alleviate sickle cell symptoms through gene editing technology Intro and outro music Garden Of Love by Pk jazz Collective  

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast Here are the highlights: ·       Loqtorzi (toripalimab-tpzi) has received FDA approval for the treatment of nasopharyngeal carcinoma, both in combination with chemotherapy and as a monotherapy, demonstrating significant improvements in progression-free survival and overall survival. ·       Vabysmo (faricimab-svoa) has been approved by the FDA as a treatment for macular edema following retinal vein occlusion, showing early and sustained vision improvement in patients, with a generally well-tolerated safety profile. ·       Omvoh (mirikizumab-mrkz) has gained FDA approval for the treatment of moderate-to-severe active ulcerative colitis, offering a targeted approach to address inflammation in this chronic inflammatory bowel disease. ·       Agamree (vamorolone) oral suspension has been approved by the FDA as a novel corticosteroid treatment for Duchenne muscular dystrophy, demonstrating efficacy in improving muscle function with a favorable safety profile. ·       Tibsovo (ivosidenib) has received FDA approval for adult patients with relapsed/refractory myelodysplastic syndromes carrying an IDH1 mutation, offering a potential treatment option for this specific group. ·       The FDA is reviewing AstraZeneca's application to allow patients or caregivers to self-administer FluMist Quadrivalent, potentially becoming the first self-administered flu vaccine, with a decision expected in the first quarter of 2024.  Intro and outro music Garden Of Love by Pk jazz Collective

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast  Here are the highlights: BIMZELX (bimekizumab) for Moderate-to-Severe Plaque Psoriasis: The FDA approved BIMZELX for treating moderate-to-severe plaque psoriasis, making it the first psoriasis treatment targeting interleukin 17A and interleukin 17F. The approval follows data from Phase 3 trials and comes after a prior delay due to COVID-related travel restrictions. Neoadjuvant Pembrolizumab for NSCLC: The FDA approved pembrolizumab for neoadjuvant and post-surgical adjuvant treatment in patients with resectable non-small cell lung cancer, adding to its indications in multiple tumor types. The approval was based on the phase 3 KEYNOTE-671 trial data. Adjuvant Nivolumab for Stage IIB/C Melanoma: The FDA granted approval to nivolumab for adjuvant treatment of melanoma in patients aged 12 and older with resected stage IIB or IIC disease, addressing the need to reduce the risk of recurrence. This is supported by the CheckMate76K trial data. Zilucoplan for Myasthenia Gravis: UCB Pharma's zilucoplan, a complement C5 inhibitor, received FDA approval for treating myasthenia gravis (MG), demonstrating rapid improvements in MG-specific efficacy outcomes based on the phase 3 RAISE study. IDP-126 (Cabtreo) First Triple-Combination Drug for Acne: Cabtreo, a triple combination topical gel for acne, received FDA approval as the first fixed-dosed, triple-combination treatment for patients aged 12 and older with acne vulgaris. Penbraya Meningococcal Vaccine in Adolescents: The FDA approved Penbraya, a vaccine covering the five most common serogroups causing meningococcal disease in adolescents, based on Phase 2 and Phase 3 trial data. It's administered as a two-dose series. Voxzogo in Dwarfism: Vosoritide (Voxzogo) was expanded for use in children under 5 with achondroplasia, the most common form of short-limbed dwarfism, after demonstrating safety and efficacy in this age group. QLOSI for blurry age-related near vision: The FDA approved QLOSI, a preservative-free eye solution, for the treatment of presbyopia, improving near visual acuity by pupil modulation and increasing depth of field. Maxigesic IV for Post-Op Pain: Maxigesic IV, a combination of paracetamol and ibuprofen, gained FDA approval for post-operative pain management, offering faster pain relief and reduced opioid usage. Xphozah for Chronic Kidney Disease: Tenapanor (Xphozah) was approved as an add-on therapy for patients with chronic kidney disease who can't tolerate or respond adequately to phosphate binders, based on phase 3 trial data, addressing high blood phosphorus levels. Zymfentra infliximab biosimilar for ulcerative colitis and Crohn's disease: Zymfentra, a subcutaneous infliximab biosimilar, received FDA approval for maintenance therapy in adults with moderately to severely active ulcerative colitis and Crohn's disease, based on LIBERTY-UC and LIBERTY-CD study findings.  Intro and outro music Garden Of Love by Pk jazz Collective    

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast Here are the highlights:  Velsipity (Etrasimod) for UC: The FDA has approved Velsipity (etrasimod) for treating moderate to severe active ulcerative colitis (UC) in adults. Etrasimod, an oral medication manufactured by Pfizer, was based on safety and efficacy data from the ELEVATE UC 52 and ELEVATE UC 12 trials, making it the second S1P class agent approved for UC in the United States. The first agent, ozanimod (Zeposia), received FDA approval in May 2021. Braftovi (encorafenib) and Mektovi (binimetinib) for NSCLC:The FDA has granted approval for the combination of encorafenib and binimetinib to treat BRAF V600E–mutant, metastatic non–small cell lung cancer (NSCLC). The approval, supported by the phase 2 PHAROS study, showed a 75% objective response rate (ORR) in treatment-naive patients. This combination was previously approved for unresectable or metastatic melanoma with a BRAF mutation. Edison System for Non-Invasive Destruction of Liver Tumors:The FDA has authorized the marketing of the HistoSonics Inc. Edison System for non-invasive destruction of liver tumors. The system utilizes a non-thermal, mechanical process of focused ultrasound to create small, vapor-filled cavities (cavitation) without heat, mechanically destroying and liquefying targeted liver tissue, including tumors. This technology is suitable for patients with sufficient functional liver reserve. Complete Response Letter for Patisiran ATTR amyloidosis:The FDA declined to approve patisiran for ATTR amyloidosis despite a favorable recommendation from advisers. The FDA noted that patisiran did not significantly improve the condition of patients with heart muscle issues or cardiomyopathy caused by ATTR amyloidosis. Patisiran, branded as Onpattro, is already approved to treat nerve damage in adult patients with hereditary ATTR amyloidosis. FDA Creates a Digital Health Advisory Committee:The FDA has established a Digital Health Advisory Committee to address complex issues related to digital health technologies, including AI/ML, augmented reality, virtual reality, and more. This committee will provide expertise and perspective to improve the FDA's understanding of the benefits, risks, and clinical outcomes associated with digital health technologies and is expected to be fully operational in 2024. Intro and outro music Garden Of Love by Pk jazz Collective  

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast Here are the highlights:  Novavax COVID Vaccine:The updated Novavax COVID-19 vaccine is approved for use in the US for those 12 and older and is a conventional vaccine containing purified, full-length recombinant Spike (rS) protein. This rS protein stimulates the immune system to generate a protective immune response, specifically by eliciting antibodies against the Spike protein of the SARS-CoV-2 virus.  Rivfloza (Nedosiran) for Primary Hyperoxaluria:The FDA approved Rivfloza, an RNA interference therapy, to treat primary hyperoxaluria type 1 (PH1) patients by lowering urinary oxalate levels. The medication will be available for eligible patients in 2024. Intravenous Cosentyx:The FDA approved an intravenous (IV) version of Cosentyx for treating adults with certain rheumatic diseases, making it the only approved IV treatment that targets interleukin-17A (IL-17A). It will be available in Q4 of 2023. Abrilada Adalimumab Biosimilar:Abrilada has been designated as interchangeable with adalimumab, meaning it can be substituted at the pharmacy level without notifying the prescribing provider. It was proven to provide similar outcomes to the reference product even when patients switched between them multiple times. Injector for Empaveli in PNH:The FDA approved the enFuse® Injector for delivering Empaveli to adults with paroxysmal nocturnal hemoglobinuria (PNH). This device is the first purely mechanical, large-volume subcutaneous drug delivery device, and Enable Injections is collaborating with other manufacturers for its application. Intro and outro music Garden Of Love by Pk jazz Collective

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast Here are the highlights: Ryzumvi (Phentolamine Ophthalmic Solution) for Dilated Pupil Reversal The FDA has approved Ryzumvi for treating pharmacologically-induced mydriasis (dilated pupils). Ryzumvi, formerly known as Nyxol, will be available in the US in 2024 and is currently the only commercially available treatment for reversing dilated eyes. Exxua (Gepirone Hydrochloride) for MDD Exxua, an antidepressant, has received FDA approval for the treatment of major depressive disorder in adults. Exxua has faced regulatory challenges in the past. It works through selective agonism of 5HT1a receptors without causing common antidepressant side effects like sexual issues or weight gain. Likmez, Liquid Formulation of Metronidazole The FDA has approved Likmez, the only liquid oral suspension of metronidazole in the US. The liquid formulation provides an alternative for patients who struggle with the tablet version due to taste or swallowing difficulties. DNA Test for Hereditary Cancers The FDA granted marketing authorization for Invitae Common Hereditary Cancers Panel, a diagnostic test identifying genetic risks for certain cancers. This is the first FDA-approved test of its kind, with over 99.0% accuracy in detecting variants in 47 cancer-associated genes. Tofidence (Actemra [toclizumab] Biosimilar) Tofidence, a biosimilar referencing Actemra, has been approved by the FDA for certain rheumatologic indications. A biosimilar, it closely resembles the FDA-approved biologic Actemra in safety, purity, and efficacy. Pombiliti + Opfolda for Pompe Disease The FDA approved Pombiliti + Opfolda, a two-component therapy for adults with Pompe disease. The therapy shows significant improvements in patient mobility and is designed to address enzyme deficiency while maintaining it's efficacy in Pompe disease. Subcutaneous Entyvio (Vedolizumab) for Moderate-to-Severe Ulcerative Colitis The FDA has approved a subcutaneous form of Entyvio for maintenance therapy in adults with ulcerative colitis. Previously approved as an intravenous formulation, this subcutaneous version achieved significant clinical remission in trials for use as maintenance therapy. Experimental Stromal Cell Platform in Mild-To-Moderate ALS The FDA Advisory committee voted against the effectiveness of BrainStorm Cell Therapeutics' NurOwn stromal cell therapy for mild to moderate ALS. BrainStorm Cell Therapeutics plans to initiate a phase 4 study in 2024. Intro and outro music Garden Of Love by Pk jazz Collective    

In this week's New FDA Approval's podcast episode, Dr. Emma Hitt Nichols discusses the latest FDA approvals from September 18, 2023 – September 22, 2023.  Please check back every Monday morning so that you can stay up to date.  Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See more details summaries and links to prescribing information at nascentmc.com/podcast Here are the highlights: Jardiance (empagliflozin) for CKD  The FDA has approved Jardiance, an SGLT2 inhibitor, for patients with end-stage kidney disease, speciifically to reduce the risk of sustained decline in eGFR, end-stage kidney disease, cardiovascular death, and hospitalization in adults with chronic kidney disease at risk of progression. The approval is based on the EMPA-KIDNEY phase 3 trial, demonstrating a significant reduction in kidney disease progression and cardiovascular death compared to a placebo.  Welireg (belzutifan) for Advanced Renal Cell Carcinoma The FDA granted Priority Review for Merck's Welireg for advanced renal cell carcinoma post other treatments. Welireg, a HIF-2α inhibitor, is being evaluated in the LITESPARK-005 trial, where it was compared with everolimus for advanced RCC treatment post PD-1/L1 or VEGF-TKI therapies. Tevimbra (tislelizumab) for Esophageal Squamous Cell Carcinoma The FDA is reviewing an application for Tevimbra for treating advanced or metastatic esophageal squamous cell carcinoma. Supported by the phase 3 RATIONALE 306 study, the drug improves overall survival rates when combined with chemotherapy in these patients. Neffy Epinephrine Nasal Spray The FDA rejected the approval of Neffy, a needle-free nasal spray for allergic reactions. Despite a positive Advisory Committee vote, the FDA demands further testing. ARS Pharmaceuticals plans to re-submit its application and appeal the decision in 2024.  Intro and outro music Garden Of Love by Pk jazz Collective

In this week's New FDA Approval's podcast episode, Dr. Emma Hitt Nichols discusses the latest FDA approvals from September 11, 2023 – September 15, 2023.  Please check back every Monday morning so that you can stay up to date.  See more details summaries and links to prescribing information at nascentmc.com/podcast Here are the highlights: • Ojjaara for Anemia in Myelofibrosis: FDA approved Ojjaara (momelotinib) for the treatment of intermediate or high-risk myelofibrosis in adults with anemia. This drug is unique as it serves both newly diagnosed and previously treated patients, addressing anemia, constitutional symptoms, and splenomegaly. Approval was based on the MOMENTUM phase 3 trial results. • Resmetirom for NASH: FDA granted priority review for resmetirom, aiming to treat non-alcoholic steatohepatitis (NASH). Madrigal, its manufacturer, seeks approval via the FDA's accelerated pathway. Resmetirom is evaluated in four phase 3 trials, including MAESTRO-NASH. The decision is set for March 14, 2024. • Updated COVID-19 Vaccines: FDA approved updated vaccines for the coming season targeting the omicron descendant XBB.1.5. While older vaccines targeted earlier strains, these new shots might provide protection against prevalent related strains. Pfizer, Moderna, and Novavax are manufacturing the vaccines. • Phenylephrine Lack of Efficacy: An FDA panel unanimously found phenylephrine, present in OTC decongestants like Sudafed and Dayquil, ineffective for its intended use. If the FDA agrees, over 250 products containing phenylephrine might be withdrawn from the market. This decision does not affect nasal preparations. • Patisiran for ATTR-CM: The FDA's advisory committee voted that the benefits of patisiran surpass its risks for treating cardiomyopathy induced by transthyretin amyloidosis (ATTR-CM). Although the drug showed small effects compared to placebo in the Phase III APOLLO-B study, it met primary and secondary endpoints. The FDA's decision will be made on or before Oct. 8, 2023. Intro and outro music Garden Of Love by Pk jazz Collective    

In this week's New FDA Approval's podcast episode, Dr. Emma Hitt Nichols discusses the latest FDA approvals from August 28, 2023 – September 8, 2023.  Please check back every Monday morning so that you can stay up to date. Here is information on the latest US FDA approvals, the week of August 28 –  September 8, 2023. Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episode at nascentmc.com/podcast  Here are the highlights: Ilaris for Gout Flares in Adults: FDA approved Ilaris (canakinumab) for adult gout flares when other treatments failed; previously approved for CAPS and other diseases. Generics of ADHD Medication: FDA approved several generic versions of Vyvanse; Takeda's patent expired; medication was in short supply due to manufacturing issues. sNDA for Balversa in mUC: Janssen submitted sNDA for Balversa for urothelial carcinoma patients with specific genetic alterations; previously granted accelerated approval in 2019. INO-3107 for RRP: FDA granted Breakthrough Therapy designation for INO-3107 for recurrent respiratory papillomatosis caused mainly by HPV-6/11; Inovio to initiate a trial in 2024. Crovalimab for PNH: FDA accepted BLA for crovalimab as a treatment for PNH based on positive Phase III study results; potential approval expected late 2023/early 2024. Intro and outro music Garden Of Love by Pk jazz Collective

Summary: In this week's New FDA Approval's podcast episode, Dr. Emma Hitt Nichols discusses the latest FDA approvals from August 21, 2023 – August  25, 2023.  Please check back every Monday morning so that you can stay up to date. See more details summaries and links to prescribing information at nascentmc.com/podcast Here are the highlights:  FDA Approves Tyruko as First Biosimilar to TysabriThe FDA approved Tyruko as the first biosimilar to Tysabri for treating relapsing forms of MS in adults. It has similar risks and side effects as Tysabri and is also approved for treating certain Crohn's Disease patients. The product is available from Sandoz Inc. Source Abrysvo RSV Maternal Immunization ApprovedThe FDA approved Abrysvo, a vaccine for preventing respiratory disease caused by RSV in infants through maternal immunization. The approval is based on Phase 3 MATISSE trial results. Abrysvo was also approved for older adults earlier this year. Source sBLA Filed for Rybrevant in Advanced NSCLCAn sBLA was filed for expanded approval of Rybrevant, combined with certain drugs, for treating advanced non–small cell lung cancer with EGFR exon 20 insertion mutations. The approval was granted to Janssen Pharmaceuticals. Source Priority Review of Xtandi in Early PCaXtandi received Priority Review by the FDA for an expanded use in early-stage prostate cancer. The sNDA is based on a Phase 3 EMBARK trial, where Xtandi showed significant benefits. The product is manufactured by Pfizer, Inc.  Intro and outro music Garden Of Love by Pk jazz Collective    

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episodes at nascentmc.com/podcast ·      Elrexfio (elranatamab-bcmm) has received accelerated approval for the treatment of adult patients with relapsed or refractory multiple myeloma who have undergone multiple prior therapies. Elrexfio is a subcutaneously delivered bispecific antibody (BsAb) immunotherapy activating T-cells to kill myeloma cells. Approval is contingent upon verification of clinical benefit in further trials. The approval was granted to Pfizer. Elrexfio joins other bispecific antibody therapies from Janssen for the same condition. ·      The FDA has approved the Hepzato Kit for delivering melphalan to the liver as a treatment for metastatic uveal melanoma (mUM) with unresectable hepatic metastases. This is the only liver-directed therapy approved for this purpose. Approval was based on the FOCUS Study (NCT02678572). The manufacturer, Delcath Systems, intends to explore broader applications beyond uveal melanoma due to the therapy's potential for other tumor types. ·      A higher-dose version of Eylea (aflibercept) injection has been FDA-approved for treating wet age-related macular degeneration (wAMD), diabetic macular edema (DME), and diabetic retinopathy (DR). This approval is based on PULSAR and PHOTON pivotal trials demonstrating non-inferior vision gains at longer dosing intervals compared to the standard dose. Eylea is a VEGF inhibitor, approved for wet AMD in 2011. The approval was granted to Regeneron Pharmaceuticals. ·      Ingrezza (valbenazine) capsules have gained FDA approval for treating chorea associated with Huntington's Disease (HD). The approval is supported by clinical studies and was granted to Neurocrine Biosciences. Ingrezza was previously approved for tardive dyskinesia. ·      The FDA has approved Sohonos (palovarotene) to treat fibrodysplasia ossificans progressive (FOP), a rare bone disease causing heterotopic ossification. The approval is based on Phase 3 MOVE trial results, showing effective reduction in bone growth. Sohonos is selective for a subtype of retinoic-acid receptors. It received Orphan Drug and Breakthrough Therapy Designations and is manufactured by Ipsen. ·      Veopoz (pozelimab-bbfg) has gained FDA approval as the first treatment for CD55-deficient protein-losing enteropathy (CHAPLE disease). CHAPLE disease is a rare inherited immune disorder causing complement hyperactivation. Veopoz is a complement inhibitor indicated for patients aged 1 year and older. It received multiple designations and was granted approval to Regeneron Pharmaceuticals. Intro and outro music Garden Of Love by Pk jazz Collective Let us know if we can help with any medical writing!  

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episodes at nascentmc.com/podcast In this week's New FDA Approval's podcast episode, Dr. Emma Hitt Nichols discusses the latest FDA approvals from August 7, 2023 – August  11, 2023.  Please check back every Monday morning so that you can stay up to date.  Here are the highlights: ·      The FDA has approved Akeega, a combination of niraparib (PARP inhibitor) and abiraterone acetate (anti-androgen), as the first orally administered treatment for BRCA-positive metastatic castration-resistant prostate cancer. The approval was based on the MAGNITUDE study showing improved progression-free survival with the addition of niraparib. ·      Talvey (talquetamab-tgvs), a bispecific T-cell engaging antibody, has received accelerated approval for patients with relapsed or refractory multiple myeloma who have undergone multiple previous therapies. ·      Izervay (avacincaptad pegol intravitreal solution), a complement C5 inhibitor, has gained FDA approval for use in geographic atrophy related to age-related macular degeneration, and showed a significant reduction in disease progression. ·      Boston Scientific's POLARx Cryoablation System has been approved for treating paroxysmal atrial fibrillation, demonstrating effectiveness in reducing paroxysmal atrial arrhythmias in the FROZEN-AF IDE clinical trial.  The full writeups are available here: https://nascentmc.com/podcast/ Intro and outro music Garden Of Love by Pk jazz Collective

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episodes at nascentmc.com/podcast In this week's New FDA Approval's podcast episode, Dr. Emma Hitt Nichols discusses the latest FDA approvals from July 31, 2023 – August  4, 2023.  Please check back every Monday morning so that you can stay up to date.  Here are the highlights: ·      The FDA has approved Zurzuvae, the first oral medication for postpartum depression, showing significant improvement in patient symptoms compared to placebo groups. ·      Jemperli, a PD-1 inhibitor, has been approved by the FDA for use with chemotherapy in adult patients with primary advanced or recurrent endometrial cancer with specific mutations. The drug is already approved for use as a single agent in similar cases and is now indicated for use in the first-line setting, with discussions for a broader indication underway with the FDA. ·      The FDA has approved the oral drug Lonsurf, in combination with bevacizumab, for the treatment of adult patients with metastatic colorectal cancer previously treated with specific chemotherapy and targetd therapies. Lonsurf, initially approved as a single agent in 2015, is also approved for advanced metastatic gastric or gastroesophageal junction adenocarcinoma. ·      The FDA has approved an expanded indication for Ervebo, a vaccine now indicated for the prevention of disease caused by Zaire ebolavirus for individuals aged 12 months and older.  Did you know, you can access our nascentmc.com/podcast page and search the notes from this podcast! So for example, if you search a term like "diabetes" it will bring up all the diabetes related therapeutic agents approved since the first episode on May 15 of this year. We're also including the prescribing information with each summary too. Check it out at nascentmc.com/podcast. Intro and outro music Garden Of Love by Pk jazz Collective  

Check out our free downloads at nascentmc.com: Implementing AMA Style – 8 Things to Get Right in Your Next Project Needs Assessments – 7 Essentials for Getting Funded Working With Your Medical Writer – 8 Ways to Get the Most out of Them See the full write ups for today's episodes at nascentmc.com/podcast Here is information on the latest US FDA approvals, the week of July 24 –  July 28, 2023. Just to summarize up front, we have RiVive receiving other the counter approval for opioid overdose; Balfaxar for urgent reversal of warfarin (VKA) therapy; Xdemvy for a common eyelid condition, Demodex blepharitis. PDUFA dates this week: Remestemcel-L for acute GVHD and zuranolone for major depressive disorder and postpartum depression.  Here are the highlights: ·       RiVive (naloxone hydrochloride nasal spray) has been approved by the FDA for over-the-counter use to treat known or suspected opioid overdose, rapidly reversing opioid effects and restoring normal respiration. This approval marks the second nonprescription naloxone product, the other being Narcan. ·       Balfaxar® (prothrombin complex concentrate, human-lans) has received FDA approval for urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist therapy in adult patients requiring urgent surgery or invasive procedures. Balfaxar® replenishes deficient clotting factors caused by warfarin therapy and was supported by the LEX-209 clinical trial, demonstrating hemostatic efficacy and non-inferiority to a comparator, Kcentra®. The medication includes a boxed warning for thromboembolic event risk. ·       XdemvyTM (lotilaner ophthalmic solution 0.25%) has been approved by the FDA as the first treatment for Demodex blepharitis, targeting inflammation caused by Demodex mites. Administered as an eye drop, Xdemvy eradicates the mites over six weeks, providing relief for patients with eyelid inflammation and discomfort. The treatment was well-tolerated, with common adverse reactions being eye stinging and burning in about 10% of patients. ·       Upcoming PDUFAs Remestemcel-L, being developed for acute graft versus host disease (aGVHD), has a PDUFA date of August 2. Derived from mesenchymal stromal cells, it may treat this life-threatening complication of bone marrow transplants. Currently, there are no approved treatments for steroid-refractory aGVHD in children under 12, making Remestemcel-L a potential solution. Zuranolone (SAGE-217) has a PDUFA date of August 5 for major depressive disorder and postpartum depression. This potential treatment, a positive allosteric modulator of GABA-A receptors, aims to rebalance dysregulated neuronal networks to help reset brain function in people with depression.  Intro and outro music Garden Of Love by Pk jazz Collective  

 In this week's New FDA Approval's podcast episode, Dr. Emma Hitt Nichols discusses the latest FDA approvals from July 17, 2023 – July 21, 2023.  Please check back every Monday morning so that you can stay up to date.  Here are the highlights:   Vanflyta® for FLT3-ITD+ AML The FDA has approved Vanflyta (quizartinib), developed by Daiichi Sankyo, for treating adult patients with acute myeloid leukemia (AML) having the FLT3-ITD mutation. The approval came after data from the QuANTUM-First trial showed improved overall survival rates when Vanflyta was added to treatment. This makes Vanflyta the third drug approved for FLT3-positive AML, joining Novartis' Rydapt® and Astellas Pharma's Xospata®.   BeyfortusTM for RSV AstraZeneca's BeyfortusTM (nirsevimab) has been approved by the FDA for preventing respiratory syncytial virus (RSV) lower respiratory tract disease in neonates, infants, and children up to 24 months of age. The approval was supported by data from three clinical trials, and the drug has also been given a fast-track designation. Beyfortus becomes the second antibody available for RSV protection in young children, following palivizumab or Synagis®. Cyfendus Anthrax Vaccine The FDA has approved CyfendusTM (Anthrax Vaccine Adsorbed, Adjuvanted) for post-exposure prophylaxis following exposure to Bacillus anthracis, the bacterium causing Anthrax, in adults aged 18 to 65. The vaccine is administered in two doses, two weeks apart, and clinical trials have found it to be non-inferior to the already available BioThrax® vaccine. Cyfendus has been in use since 2019 under a pre-emergency use authorization. YcanthTM for Molluscum Contagiosum The FDA has approved Verrica Pharmaceuticals' YcanthTM (cantharidin) topical solution for treating molluscum contagiosum in patients aged 2 and older. The approval is based on results from two identical phase 3 trials involving over 500 patients. Despite being available via compounding sources for years, Ycanth is the first FDA-approved standardized product for molluscum contagiosum. This podcast is brought to you by Nascent Medical. If you're a project manager at a CME or medical communications agency and need on-call medical writing assistance please visit Nascent Medical. We are a team of MD- and PhD-level medical writers and can create slide decks, white papers, ad board summaries, manuscripts, needs assessments, and much more. We also do medical editing using AMA style and factchecking. Visit Nascent Medical. Intro and outro music Garden Of Love by Pk jazz Collective

In this week's New FDA Approval's podcast episode, Dr. Emma Hitt Nichols discusses the latest FDA approvals from July 10, 2023 – July 14, 2023.  Please check back every Monday morning so that you can stay up to date.   Get our free download! Implementing AMA Style – 8 Things to Get Right  Here are the highlights of this week's episode: Opill® is the first over-the-counter birth control pill approved by the FDA, containing norgestrel tablets as a progestin-only contraceptive. Opill® thickens cervical mucus, prevents the release of eggs, and thins the uterine lining to provide effective contraception. Opill® will be available in stores in the US in early 2024 Leqvio® (inclisiran) has received FDA approval for expanded use in patients with elevated LDL-C and increased risk of heart disease, in addition to its previous indications. The drug is an RNA therapy that lowers LDL-C levels and is administered via a single subcutaneous injection initially, followed by repeat doses every 3 and 6 months. Clinical studies showed a mean reduction of approximately 50% in LDL-C levels; common adverse reactions are injection site reactions, arthralgia, and bronchitis. Takeda has withdrawn its Biologics License Application for the Dengue vaccine candidate, TAK-003, following FDA's request for additional data not included in the application. TAK-003, also known as Qdenga®, is already approved in various countries. Dengue is a mosquito-borne disease causing significant illness and deaths worldwide; currently, there are limited vaccines or other preventive agents available. This podcast is brought to you by Nascent Medical. If you're a project manager at a CME or medical communications agency and need on-call medical writing assistance please visit Nascent Medical. We are a team of MD- and PhD-level medical writers and can create slide decks, white papers, ad board summaries, manuscripts, needs assessments, and much more. We also do medical editing using AMA style and factchecking. Visit nascentmc.com Intro and outro music Garden Of Love by Pk jazz Collective