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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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