Sarepta CRL Shines Light on FDA’s Review of DMD Treatment

A routine document released by the FDA raised new questions about the agency’s rejection — and subsequent approval — of Sarepta’s Duchenne muscular dystrophy (DMD) treatment Vydonos 53.

The FDA released a complete response letter sent to Sarepta last August, which cited the risk of infections and renal toxicity seen in pre-clinical models.

The CRL’s release sparked a flurry of speculation over the agency’s quick reversal just months later in approving the drug.