Sep 19th - EMA decides not to renew Translarna (ataluren - sold by PTC Therapeutics) after conditional approval was granted over 9 years ago. Click on Translarna above for the link (I hope).
Translarna is a treatment for ambulatory DMD boys with the nonsense mutation (15%).
It has taken that long to complete a phase 3 confirmatory trial. The results showed efficacy in the overall group of patients (359 boys), but failed to show efficacy in their primary analysis population (subset) which was their chosen endpoint benchmark (boys who were able to walk at least 300m in 6 minutes and stay in a standing position for more than 5 secs before the trial). The trial measured the distance walked in 6 minutes.
Sep 19th - EMA decides not to renew Translarna (ataluren - sold...
-
- There are more pages in this discussion • 427 more messages in this thread...
You’re viewing a single post only. To view the entire thread just sign in or Join Now (FREE)
