ARTICLE | Regulation

Sarepta, FDA have ‘alignment’ to evaluate DMD gene therapy on accelerated pathway

Fall submission to include biomarker data company believes is predictive of clinical benefit, as well as intermediate functional data

July 29, 2022 8:50 PM UTC

Sarepta will once again test FDA’s willingness to grant accelerated approval to a DMD therapy based on a combination of biomarker evidence and a limited set of functional data, even as the company’s better-powered study of the same gene therapy is set to read out in mid-2023.

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said Friday it intends to submit a BLA to FDA for micro-dystrophin gene therapy delandistrogene moxeparvovec (SRP-9001) in the fall, rather than wait for data next year from the Phase III EMBARK trial to treat Duchenne muscular dystrophy. It is developing the therapy with Roche (SIX:ROG; OTCQX:RHHBY)...

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