Beqvez for Hemophilia B, Anktiva for Bladder Cancer, Pivya for UTI, Tovorafenib for Pediatric Low-Grade Glioma, Entyvio Maintenance for Crohn’s Disease, Alecensa for NSCLC, SPG601 for Fragile X Syndrome, Lumisight for Visualizing Breast Cancer

New FDA Approvals

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.