FDA Staff Had Inclination Towards Rejecting Sarepta Duchenne Gene Therapy

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  • 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.
Posted In: BiotechNewsHealth CareFDAMediaGeneralBriefs
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