Posted by Michael Wonder on 09 Mar 2017
PTC Therapeutics announces FDA acknowledgment of new drug application filing for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy
6 March 2017 - FDA has assigned a PDUFA date of 24 October 2017.
PTC Therapeutics today announced that the U.S. FDA has acknowledged the filing over protest of PTC's new drug application for Translarna (ataluren), an oral, first-in-class, protein restoration therapy for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD).
The Company is seeking approval to market the drug for the treatment of nmDMD patients in the United States. Translarna received marketing authorisation for patients with nmDMD in the European Union in August 2014 and is now available in over 25 countries.
The FDA has granted standard review and assigned a Prescription Drug User Fee Act (PDUFA) date of 24 October 2017. The PDUFA date is the target date for the FDA to complete its review of the NDA.
