(Reuters) – Shares of Sarepta Therapeutics soared 29% pre-market on Monday as backing from advisers to the US health regulator increased certainty of an accelerated approval of the gene therapy of the company for a genetic muscle wasting disorder.
Eight of 14 Food and Drug Administration advisers voted late Friday that the company had enough data to support an accelerated approval of its gene therapy for Duchenne muscular dystrophy (DMD).
“We are increasingly optimistic that SRP-9001 will receive expedited approval, especially as the panel voted in line with how we believe the agency’s senior leadership views the application,” said Joseph Schwartz, an analyst at SVB Securities.
The vote came after FDA staff raised concerns early last week that data from the company’s mid-stage trial lacked “unequivocal evidence” for the therapy’s benefits.
TD Cowen analyst Ritu Baral expects an approval for the therapy, especially considering the interest of FDA leadership in promoting accelerated biomarker-based approvals for gene therapies.
Senior FDA official Peter Marks said in March that the regulator is moving to encourage the use of disease-related biomarkers that can predict the efficacy of gene therapies for diseases with small patient populations rather than waiting for proof. definitive benefit to the patient.
The FDA, which often follows the advice of its expert advisors but is not required to, plans to make a decision on expedited approval by May 29.
“There was little chance that the vote would have been positive were it not for the commitment of patient advocates throughout the development and review process,” William Blair analyst Tim Lugo said in a note on Monday, describing the close nature of Friday’s vote.
Shares of the company rose to $154.92 in premarket trading. Trading in shares was halted on Friday due to the meeting.
(Reporting by Aditya Samal; Editing by Sriraj Kalluvila)