FDA Staff Had Inclination Towards Rejecting Sarepta Duchenne Gene Therapy

Reviewers at the FDA were leaning toward rejecting Sarepta Therapeutics Inc's SRPT closely watched gene therapy for Duchenne muscular dystrophy.
A top official had to intervene earlier this year, Stat News reported citing three people with direct knowledge of the agency deliberations.
Inside the FDA office that oversees gene therapies, some staff had reached a non-binding conclusion that Sarepta's gene therapy should be rejected, the individuals said.
Peter Marks, a top FDA official, stepped in and directed staff to schedule an advisory committee meeting on the therapy on May 12.
The FDA seeks to optimize the development of gene therapies to help get accelerated approval of therapies by support using biomarkers as substitutes for other biological indicators in gene-therapy clinical trials.
The advisory committee meeting will be hosted as a virtual meeting. SRP-9001 is Sarepta's investigational gene therapy for treating Duchenne muscular dystrophy.
The FDA took a U-turn when it announced to hold an advisory committee meeting related to Sarepta's SRP-9001 (delandistrogene moxeparvovec) marketing application.
In its Q4 earnings release, Sarepta said the mid-cycle meeting is complete, and FDA does not plan to hold an advisory committee for SRP-9001.
Price Action: SRPT shares are down 5.75% at $129.76 on the last check Thursday.