Duchenne Muscular Dystrophy Treatment Narrowly Captures Support of FDA Advisors
FDA advisors narrowly supported accelerated approval for Sarepta Therapeutics’ investigational gene therapy for Duchenne muscular dystrophy (DMD) on Friday. In a 8-to-6 vote, the agency’s Cellular, Tissue, and Gene Therapies Advisory Committee said the overall benefits and risks supported SRP-9001 (delandistrogene moxeparvovec) for ambulatory Duchenne patients, using expression of Sarepta’s micro-dystrophin as a surrogate endpoint. […]
FDA OKs Sparsentan for IgA Nephropathy-Related Proteinuria
The FDA granted accelerated approval to sparsentan (Filspari) for proteinuria in IgA nephropathy, Travere Therapeutics announced, marking the first non-immunosuppressive therapy for this rare condition. Also known as Berger’s disease, proteinuria in IgA nephropathy is the leading cause of kidney failure due to glomerular disease, and sparsentan is specifically indicated to reduce proteinuria in adults […]