FDA staff unconvinced by Sarepta’s muscle wasting drug
By Natalie Grover
(Reuters) – Sarepta Therapeutics Inc lost more than half its market value on Friday after U.S. Food and Drug Administration staff said they were unconvinced about the effectiveness of the company’s drug for a rare muscle wasting disorder.
The FDA report comes a day after the U.S. regulator rejected BioMarin Pharmaceutical Inc’s rival drug Kyndrisa.
Sarepta’s drug, eteplirsen, is designed to treat a subset of patients with Duchenne muscular dystrophy (DMD), an incurable fatal disease, that hampers muscle movement and affects one in 3,600 newborn boys, with most patients dying by the age of 30.
There are no FDA-approved drugs for DMD, and pressure has been mounting on the U.S. regulator to quickly approve treatments.
In its report on Friday, the FDA’s staff reviewers said it was prepared to be flexible with respect to the devastating illness, but would not sanction approval without established efficacy.
The FDA staff questioned the validity of the ‘small’ study used by Sarepta to test its drug’s efficacy, saying they considered the trial merely ‘exploratory’ because of its design. (http:1.usa.gov/1RKkQ3q)
Eteplirsen, like Kyndrisa, skips a faulty section of the gene to produce dystrophin, the lack of which is believed to cause DMD.
Both drugs target a particular subset of DMD patients, which translates into an addressable population of about 1,800 boys in the United States and about 5,000 outside, according to Wedbush Securities analyst Heather Behanna.
The FDA documents on eteplirsen were published earlier than expected. An advisory committee to the FDA is expected to make a recommendation on the drug on Jan. 22.
Sarepta’s shares were down 58 percent at $13.43 in heavy early trading on Friday.
(Reporting by Natalie Grover in Bengaluru; Editing by Saumyadeb Chakrabarty)
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