2023-06-22 14:21:29 ET
While the US FDA on Thursday granted accelerated approval to Sarepta Therapeutics' ( NASDAQ: SRPT ) latest Duchenne muscular dystrophy treatment SRP-9001, also known as Elevidys, the label is narrower than the company had hoped for.
The agency approved the one-time gene therapy, also known as delandistrogene moxeparvovec, for boys four and five years old with DMD and a confirmed mutation in the DMD gene.
Sarepta said in a statement that continued approval for the treatment may be contingent upon verification and description of benefit in confirmatory studies.
An FDA advisory committee in May endorsed the treatment by a vote of 8-6. However, later that month, the agency said it needed more time to review the application.
Shares of Sarepta were halted Thursday afternoon due to pending news.
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Sarepta Duchenne treatment wins FDA nod, but with smaller population