Snubbed at the FDA, PTC files their three-time loser for Duchenne MD over protest
PTC Therapeutics $PTCT isn’t going to let a slate of three consecutive, failed studies for ataluren block its review at the FDA. Using the agency’s rules for applications on drugs regulators have refused to file, the biotech says it filed over protest, using regulatory procedures to force a PDUFA date of October 24.
PTC CEO Stuart Peltz was frank about why the FDA wouldn’t accept their application early last year. Regulators felt that the company had flat failed to make a case for the drug that was worthy of a review. And he’s been beating on the FDA’s door ever since, despite the agency’s decision to reject its first appeal, claiming once again that the “totality” of the data warrant its approval.
Unlock this article instantly by becoming a free subscriber.
You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.