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The FDA’s once-raging internal debate over Sarepta Therapeutics has cooled, but a recently unearthed document might portend failure for the next company angling to win an approval in Duchenne muscular dystrophy.

PTC Therapeutics is seeking approval for its treatment, ataluren, despite the drug’s repeated clinical failures and the FDA’s 2016 refusal to even consider the drug. Approval would seem unlikely, and it looks to be even more of a reach in light of a memo written by Dr. John Jenkins, an FDA official, back in the heat of the Sarepta saga.


The memo from Jenkins came in the form of a letter to the FDA’s commissioner at the time, Dr. Robert Califf, to lay out his concerns about the Sarepta approval. In it, Jenkins makes reference to two other DMD treatments: BioMarin’s rejected drisapersen and another drug, whose name was redacted — but whose description matches up with PTC’s product.

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