Sarepta on watch as FDA comments on gene therapy ahead of AdCom meeting
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Sarepta Therapeutics (NASDAQ:SRPT) fell ~2% pre-market Wednesday after the FDA posted briefing documents ahead of an advisory committee meeting on May 12, 2023, regarding the company's marketing application for gene therapy SRP-9001.
In April, Sarepta (SRPT) shares sank after the FDA's Office of Therapeutics decided to convene an AdCom meeting ahead of a regulatory action date in late May for the company's biologics license application for SRP-9001 targeted at Duchenne muscular dystrophy.
Days later, Sarepta (SRPT) and its partner for the treatment Catalent (CTLT) came under pressure after media reports indicated that some staff members at the FDA initially decided to reject the therapy if not for the intervention of a top official.
In the briefing documents, the FDA has reiterated its concerns about SRP-9001 with regard to its efficacy and safety.
"FDA notes that the clinical studies conducted to date do not provide unambiguous evidence that SRP-9001 is likely beneficial for ambulatory patients with DMD," the officials wrote,
The agency "has safety concerns related to the possibility of administering an ineffective gene therapy," they added.
The FDA's advisory committees issue non-binding recommendations. However, the regulator usually follows them before making a final decision on authorizations.