Scholar Rock has removed the OAI-classified Catalent Indiana facility from apitegromab’s US filing, preserving its September 30 FDA target through an alternate manufacturer while withdrawing and preparing to resubmit the European application.
FDA has accepted Teva’s ecopipam NDA with Priority Review after Phase 3 treatment reduced the risk of tic relapse by 53% among pediatric responders.
Werewolf and Ambros secure $150m to fund neridronate through its pivotal CRPS-1 Phase 3 readout and planned FDA filing. See what is at stake.
EU approval moves TECVAYLI plus daratumumab into earlier multiple myeloma treatment after Phase 3 data cut progression risk by 83.4%.
China’s Center for Drug Evaluation has accepted opamtistomig for previously treated advanced EP-NEC and placed the application under Priority Review, while detailed pivotal data await presentation.
LUCIDITY met its FDA-agreed primary endpoint with a 55% lower composite rate of Level 2 and Level 3 hypoglycemic events, supporting an NDA planned by year-end.
FDA approval gives adults with fibrodysplasia ossificans progressiva another treatment option after OPTIMA recorded just one to two new bone lesions with Pasatru versus 19 with placebo over 56 weeks.
Preliminary Phase 1 data showed brain exposure, a half-life above 30 hours and dose-dependent IL-1β inhibition for AC Immune’s oral NLRP3 inhibitor ACI-19764.
FDA accelerated approval makes Genglycos the first treatment for GSDIa, based on a 31% reduction in daily cornstarch intake that must now be confirmed through long-term follow-up.
INTerpath-001 met recurrence-free and distant metastasis-free survival endpoints in 1,137 patients, giving individualized mRNA cancer therapy its first positive Phase 3 readout.