2023-05-12 08:27:11 ET
Sarepta Therapeutics ( NASDAQ: SRPT ) shares are on hold for trading as a group of independent experts at the FDA is scheduled to meet on Friday to weigh in on its marketing application for gene therapy SRP-9001 targeted at Duchenne muscular dystrophy.
The panelists of the FDA's Cellular, Tissue, and Gene Therapies Advisory Committee are expected to vote on the treatment's approvability at the end of the meeting.
In April, Sarepta ( SRPT ), which has partnered with Roche ( OTCQX:RHHBY ) ( OTCQX:RHHBF ) for SRP-9001, came under pressure after announcing that FDA has scheduled an AdCom meeting to discuss its biologics license application.
According to Stat News , Dr. Peter Marks, the head of the FDA's biologics unit who has backed accelerated approvals for gene therapies, intervened to hold an AdCom meeting while some FDA staff members initially decided to reject the treatment.
Releasing briefing documents ahead of the meeting on Wednesday, the FDA reiterated its concerns about SRP-9001 in relation to its efficacy and safety.
While the recommendations of FDA's AdCom panels are non-binding, the regulator usually follows them before making a final decision which in the case of SRP-9001 is due before May 29, 2023.
Given the high stakes of the decision, several investment firms tried to forecast the outcome of the meeting.
Noting that there are only two hours of discussion before the voting, Bank of America analyst Neena Bitritto-Garg expects the vote to be one-sided.
"Given that there will be ~2 hours of discussion prior to the vote tomorrow, during which panelists can align, we expect the vote to ultimately be onesided," Bitritto-Garg, who has a Buy rating and a $170 per share target on SRPT wrote.
SVB Securities analyst Joseph Schwartz argues that the FDA briefing documents on SRP-9001 set an uncertain backdrop for the panelists, while Evercore ISI analysts project a 50%-60% chance of approval.
More on SRP-9001
- Sarepta on watch as FDA comments on gene therapy ahead of AdCom meeting
- Sarepta's Prospects For SRP-9001 Approval Remain Strong Despite AdCom Setback
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Sarepta shares on hold for trading ahead of closely watched FDA meeting