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PTC Therapeutics (PTCT) failed to convince a panel of FDA-appointed experts that its Duchenne muscular dystrophy drug Translarna was worthy of approval.

By a 10-1 vote, the FDA advisory panel concluded that while Translarna might be effective, the data submitted by PTC were inconclusive, and more work was needed to establish the drug’s efficacy. The sole dissenting vote to approve the drug now came from a panel member with a child with DMD.


The negative panel vote will likely lead to the FDA rejecting Translarna, asking PTC to complete a new clinical trial before resubmitting for approval again. The FDA has until October 24 to make the final decision.

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