How Accelerated Approval Works – And How It Doesn't
PTC Therapeutics argues FDA did not apply the same standard to Translarna in Duchenne muscular dystrophy as previous DMD products, but FDA says accelerated approval was not possible because of negative efficacy data.
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Use of expedited pathway based on drug’s effect on brain amyloid would be problematic given advisory committee’s views of a disconnect between biomarker data and clinical efficacy results; however, one FDA expert sees potential for agency to grant accelerated approval on basis of an intermediate clinical endpoint – the same endpoint on which Biogen seeks regular approval.
PTC Therapeutics appears to be hoping that CDER Director Woodcock will do for ataluren what she did for Sarepta's exon-skipping Duchenne muscular dystrophy treatment Exondys 51 since dispute resolution request would first go to Office of New Drugs, where Woodcock is acting director.
US FDA officials say they need knowledge of the disease to have the confidence to use accelerated approval.