FDA staff forces advisory panel vote on Sarepta muscle drug

Sarepta Therapeutics Inc’s muscle wasting drug got a new lease of life on Friday after staff at the U.S. Food and Drug Administration made it necessary that an independent panel of experts on Monday make a concrete recommendation on whether the drug should be approved or not.

The staff reviewers on Thursday issued a scathing review on the drug and had asked the independent panelists only for feedback on Sarepta’s trial data, without seeking their recommendation through voting questions.

On Friday, they asked that the independent panelists vote on five questions, the most significant being whether the trial data demonstrated substantial evidence that the drug, eteplirsen, was effective. (1.usa.gov/23Od3YH)

Sarepta’s stock surged 30 percent in afternoon trading. They had closed down about 44 percent on Thursday.

The fact that there are voting questions is encouraging to an extent, but some of the questions seem designed to set Sarepta up for failure, said Janney Montgomery Scott’s Debjit Chattopadhyay.

Panelists on Monday will make their recommendations after taking into account the FDA staff review, Sarepta’s analysis, and public commentary. While the FDA is not obligated to follow the panel’s advice, it typically does.

Sarepta is seeking accelerated approval for eteplirsen, which is designed to treat a subset of patients with Duchenne muscular dystrophy (DMD). DMD has no FDA-approved treatments.

Pressure has been mounting on the FDA to approve treatments for the progressive, muscle wasting disease. Most patients die by the age of 30.

A similar drug developed by BioMarin Pharmaceutical Inc was rejected in January, without any clear cut voting questions in the panel review.

The head of the FDA’s pharmaceuticals division is expected to attend the independent panelists’ meeting on Monday, indicating an unusually high degree of scrutiny on the drug and the disease in general.

The FDA has also set aside about two-and-a-half hours for comments from doctors, patients, advocates of the drug and others. Such meetings usually have one hour assigned for such public comments.

(Reporting by Natalie Grover in Bengaluru; Editing by Savio D’Souza)

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