Disparities in FDA decisions on Sarepta DMD therapy raise questions about integrity of review process

Documents released Wednesday by FDA reveal unexplained discrepancies between an August complete response letter for Vyondys 53 golodirsen and December’s surprising accelerated approval of the therapy. The differences between FDA’s skepticism about the development program and its decision to approve the Sarepta Therapeutics Inc. (NASDAQ:SRPT) drug raise questions about the integrity of the agency’s review process.

Following the complete response letter (CRL) on Aug. 19, Sarepta submitted a Formal Dispute Resolution Request on Sept. 13, in which it disagreed with FDA’s conclusion that Vyondys would only lead to a small clinical benefit, and argued that the agency failed to recognize the therapeutic context of “dire need” in which Vyondys would be used. The company also asserted that the safety risks were overstated. The company then discussed those issues with FDA in an Oct. 17 meeting...