- PTC Therapeutics (NASDAQ:PTCT) slumps 14% premarket on increased volume in apparent response to the release of briefing documents ahead of Thursday's FDA Advisory Committee review of its marketing application for Duchenne muscular dystrophy (DMD) candidate Translarna (ataluren).
- This a rare case of an advisory committee review of an application "filed-over-protest" in which the applicant insists that the FDA further review the data that was initially regarded as insufficient to support approval. On two prior occasions, 2011 and 2016, the FDA rejected the company's application under "Refuse to File" which means the filing was not complete enough to permit a substantive review.
- The present situation is the result of the FDA's controversial approval of Sarepta Therapeutics' (NASDAQ:SRPT) application for EXONDYS 51 in September 2016 that many observers perceived lacked sufficient proof that the drug works.
- FDA briefing doc
- Addendum to FDA briefing doc
- Errata to FDA briefing doc
- PTC briefing doc
- Addendum to PTC briefing doc
- Now read: Sarepta Belongs Closer To After Golodirsen Data
Original article