Sarepta's once-rejected drug gets a second FDA opinion
In August, the FDA rejected Sarepta Therapeutics’ second Duchenne muscular dystrophy drug for reasons that were more than a little unclear. Last night, the agency changed its mind entirely and offered little in the way of explanation.
The FDA approved Vyondys 53 to treat the roughly 8% of Duchenne patients whose disease results from a specific genetic error. The agency’s statement made no mention of the prior rejection, but Sarepta said in a news release that it had filed an appeal with the FDA and worked to address regulators’ concerns.
As for cost, Sarepta said it will price Vyondys 53 “at parity” with its previous Duchenne treatment, Exondys 51, which is dosed based upon patients’ weight and can cost upward of $1 million per year.
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The FDA approved Vyondys 53 to treat the roughly 8% of Duchenne patients whose disease results from a specific genetic error. The agency’s statement made no mention of the prior rejection, but Sarepta said in a news release that it had filed an appeal with the FDA and worked to address regulators’ concerns.
As for cost, Sarepta said it will price Vyondys 53 “at parity” with its previous Duchenne treatment, Exondys 51, which is dosed based upon patients’ weight and can cost upward of $1 million per year.
Read more.
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