Clinical Trial News

ConSynance Therapeutics receives FDA Rare Pediatric Disease Designation for CSTI-500 in Prader-Willi Syndrome, aiming to address hyperphagia and severe temper outbursts through a Triple Monoamine Reuptake Inhibitor.

Bio-Thera's BAT2206, a biosimilar to Stelara®, has its BLA accepted by the FDA and MAA by the EMA, marking a milestone as the first ustekinumab biosimilar from a Chinese company submitted for approval in the US and EU. The applications are supported by comprehensive analytical, non-clinical, and clinical data.

Spectral Medical Inc. updates on Tigris trial, a Phase 3 study for Polymyxin B Hemoperfusion in treating endotoxemia and septic shock, with 101 patients enrolled. Aiming for full enrollment, the company hosted an Investigator Meeting to support trial sites. PMX, Spectral's therapeutic device, targets septic shock treatment, with FDA Breakthrough Device Designation.

Indiana University researchers, led by Renzhi Han, developed a novel gene therapy using a triple-adeno-associated virus vector system to restore full-length dystrophin protein in Duchenne muscular dystrophy (DMD) mice models, improving muscle health and strength. This approach, promising better outcomes than current treatments, is advancing toward clinical development.

Glecirasib, a KRAS G12C inhibitor by Jacobio Pharmaceuticals, shows promise in treating KRAS G12C mutant cancers with high selectivity and low gastrointestinal toxicity. Clinical trials demonstrate significant efficacy in NSCLC and pancreatic cancer, with ongoing studies exploring its potential in various solid tumors and combination therapies.

FDA approves Femlyv, the first orally disintegrating birth control pill, on July 22, 2024, providing an alternative for individuals with difficulty swallowing medication.

Xeomin, first approved in 2011 for frown lines, now also treats forehead and crow's feet lines. It's the first FDA-approved neurotoxin for simultaneous treatment of upper facial lines, containing only essential ingredients. The new indication is based on clinical studies showing its efficacy and safety, with high patient satisfaction and continued efficacy over multiple treatment cycles.

Semaglutide, known as Wegovy, approved for preventing heart attacks and strokes in overweight individuals with cardiovascular disease. It reduces major cardiovascular events by 20%, aids weight management, and is under review for NHS use. Novo Nordisk faces supply constraints amidst high demand.

The FDA has accepted the resubmission of a biologics license application (BLA) for remestemcel-L, aimed at treating pediatric patients with steroid-refractory acute graft-vs-host-disease (SR-aGVHD). This follows previous submissions and addresses chemistry, manufacturing, and control issues, with clinical data from the phase 3 MSB-GVHD001 trial supporting the application.

A single HIV viral load test may yield false positives for those on long-acting injectable cabotegravir (CAB-LA) PrEP, according to a multi-country study. A second test with a new blood sample reliably distinguishes true from false positives. Presented at the 2024 International AIDS Conference, the study highlights the need for optimizing HIV testing methods for CAB-LA PrEP users.