New FDA Approvals Emma Hitt Nichols, PhD

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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.
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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.
 

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•    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.

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- **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.
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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.  

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- 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.
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