Sarepta Plunges As FDA Takes Surprise Turn; AdComm Meeting Set For Duchenne Gene Therapy

  • The FDA will hold an advisory committee meeting related to Sarepta Therapeutics Inc's SRPT SRP-9001 (delandistrogene moxeparvovec) marketing application.
  • SRP-9001 is Sarepta's investigational gene therapy for treating Duchenne muscular dystrophy, developed in partnership with Roche Holdings AG RHHBY.
  • "This change adds drama to the equation," said Baird analyst Brian Skorney in a Reuters report. He added that the FDA's inconsistency might signal more internal debate than anticipated.
  • A date has not been set for the adcomm meeting, but it will be held sometime before May 29, granted as the PDUFA date under priority review status
  • 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.
  • The meeting will likely focus on the clinical trial's surrogate endpoint, biomarkers, and approaches like accelerated approval to advance cell and gene therapies.
  • Duchenne is characterized by a mutation in the dystrophin gene that results in the lack of dystrophin, which acts as a shock absorber for muscle at the membrane. 
  • SRP-9001 is designed to treat the proximate cause of Duchenne by delivering to the muscle a gene that codes for a shortened, functional form of dystrophin.
  • Credit Suisse analyst maintains Sarepta Therapeutics with a Neutral rating, lowering the price target from $144 to $139.
  • Price Action: SRPT shares are down 20.27% at $119.33 on the last check Friday.
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