Sarepta to submit NDA for DMD candidate by YE

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said it plans to complete submission of a rolling NDA to FDA by year end for golodirsen (SRP-4053) to treat Duchenne muscular dystrophy amenable to exon 53 skipping. The company said it will seek accelerated approval for golodirsen based on an increase in dystrophin protein as a surrogate endpoint. The announcement follows a February meeting with FDA's Division of Neurology Products.

In 2016, FDA approved Sarepta's Exondys 51 eteplirsen to treat DMD amenable to exon 51 skipping. The agency based its approval of Exondys 51 on the same surrogate endpoint of an increase in dystrophin. Exondys 51 is a phosphorodiamidate morpholino (PMO) that induces skipping of exon 51 dystrophin mRNA (see BioCentury, Oct. 13, 2017)...