Duchenne approval exposes FDA rift over Sarepta gene therapy

With a controversial decision that goes against his staff’s advice, Peter Marks, head of the FDA’s Center for Biologics Evaluation and Research, has granted approval to Sarepta Therapeutics’ treatment for Duchenne muscular dystrophy, Elevidys (Delandistrogene Moxeparvovec-rokl). Elevidys is based on delivering a smaller but functional form of dystrophin (micro-dystrophin) into muscle cells using an Adeno-associated viral system (AAV). Elevidys, previously available on accelerated approval for boys 4 or 5 years of age only, is now fully approved for 80% of Duchenne patients in the U.S. Despite patient advocacy groups supporting the FDA’s decision, several review teams have rejected Serepta’s application due to conflicting data.