FDA Forces Panel to Vote on Muscle Drug Sarepta

Sarepta Therapeutics’ drug to treat a muscle wasting disease was given a new lifeline on Friday after the U.S. Food and Drug Administration forced a panel of independent experts to make a definite recommendation on if the drug should be or should not be approved.

On Thursday, the reviewers from the FDA issued a critical review on the drug and asked the panelists only for any feedback on the trial data of Sarepta, asking for their recommendations through voting.

However, on Friday, the same staffers from the FDA asked that the group of independent panelists take a vote based on five questions, with the most significant if the trial data had demonstrated sufficient evidence the drug, called eteplirsen, had been effective.

Stock at Sarepta was up by 30% in trading during the afternoon, after it had closed 44% down on Thursday.

Because there were voting questions involved it was encouraging to some extent, but some questions seemed to be designed to set up Sarepta for failure said an online analyst.

On Monday, panelists will make recommendations after they take into account the staff review by the FDA, the analysis by Sarepta and the public’s commentary.

While the FDA has not obligation to follow the advice of the independent panel of experts, it typically will.

Sarepta is looking for accelerated approval for its drug, which has been designed for treatment of patients that have Duchenne muscular dystrophy or DMD. There currently are no treatments approved by the FDA for DMD.

Pressure has mounted on the U.S. regulator to approve a treatment or more than one for the progressive disease that is muscle wasting with most patients dying before they reach 30.

A drug that is similar that BioMarin Pharmaceutical developed was rejected this past January by the FDA without a single clear cut voting question.

The head of the pharmaceutical division of the FDA is expected to be in attendance for the panelist’s meeting Monday, indicating the high degree of scrutiny the new drug and the disease is getting in general.



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