In any other circumstance, the US FDA's Peripheral & CNS Drugs Advisory Committee’s Sept. 28 meeting on PTC Therapeutics Inc.’s ataluren would have been one that most sponsors could afford to skip since the briefing documents made clear the agency didn't believe ataluren’s efficacy had been demonstrated in the two trials completed by PTC to date.
PTC isn’t the first sponsor to try the equivalent of a Hail Mary pass to save an application by forcing a public advisory committee review
Ataluren Advisory Committee Coverage
From breaking down the questions and the committee roster before the meeting to tracking the aftermath in the patient community, the Pink Sheet offered a variety of stories about the ataluren advisory committee:
- Mechanism-Based Drug Rejection? Ataluren’s Unlikely Personalized Medicine Milestone
- Duchenne Community Keeps The Faith After Dual New Product Setback
- Cystic Fibrosis Failure Weakens PTC's Duchenne Argument, FDA Says
- How Accelerated Approval Works – And How It Doesn't
- Patients Can't Clear Translarna's Data Hurdles As PTC Falls Short At FDA Panel
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