FDA oks Sarepta muscular dystrophy drug after previous rejection
Dec. 13, 2019
The U.S. FDA granted an accelerated approval to Sarepta Therapeutics' therapy for Duchenne muscular dystrophy, reversing its original rejection.
The agency turned down Sarepta’s Vyondys 53 in August for safety reasons. After receiving a complete response letter, the drugmaker made a formal dispute resolution request and was able to resolve matters raised in the CRL.
The drug is an antisense oligonucleotide from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform.
The drugmaker says commercial distribution in the U.S. will commence immediately.
Read the press release
