Sarepta Therapeutics, Inc. (SRPT - Free Report) announced that it has been requested by the FDA to provide dystrophin data from biopsies obtained from the ongoing confirmatory study on eteplirsen, as part of the agency’s ongoing evaluation of the New Drug Application (NDA).
The company said that it plans to submit the data from 13 patient biopsy samples to the FDA over the next few weeks to facilitate the agency’s decision on the NDA.
Sarepta is looking to get eteplirsen approved for the treatment of patients suffering from Duchenne muscular dystrophy (DMD), amenable to exon 51 skipping.
We note that last month, the company received a notice from the FDA, which stated that the agency will not be able to deliver a decision regarding the approval status of eteplirsen on the previously scheduled date of May 26 and has thus, extended the Prescription Drug User Fee Act (PDUFA) date. The agency informed the company that it continues to review the NDA and will try to reach a decision as early as possible.
We remind investors that DMD drugs have not witnessed much success on the regulatory front so far. Last week, BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that it does not intend to continue with the development of Kyndrisa. The company withdrew its marketing application in the EU following discussions with the Committee for Medicinal Products for Human Use (CHMP), which indicated that the drug would receive a negative opinion from the committee. Kyndrisa had also received a complete response letter (CRL) earlier this year.
DMD, a devastating and debilitating disease, is estimated to affect nearly 1 in every 3,500–5,000 boys born across the world, indicating significant unmet need for treatments in this space.
Investor focus is expected to remain on the outcome of the approval status of eteplirsen.
Sarepta currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the health care sector include Bristol-Myers Squibb Company (BMY - Free Report) and Pfizer Inc. (PFE - Free Report) , both sporting a Zacks Rank #1 (Strong Buy).
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