The US Food and Drug Administration (FDA) has recently agreed to review PTC Therapeutics’ licensing application for Duchenne drug, Translarna. The FDA is expected to complete its review by 24 October 2017.
PTC first applied to the FDA in January 2016 but this application was rejected. The FDA said that it was not sufficiently complete to review. This led to an appeal by PTC, which was again rejected by the FDA in October 2016.
The company then escalated its appeal to the next supervisory levels of the FDA, which has now resulted in the application being accepted for review.
Translarna received marketing authorisation for patients with nonsense mutations in the European Union in August 2014 and is currently available in the UK.
Dr Stuart Peltz, Chief Executive Officer of PTC Therapeutics, said:
We look forward to working closely with the FDA and the DMD community to bring this much-needed therapy to patients. We believe that the totality of clinical data in our New Drug Application (NDA), which includes the results of two of the largest placebo-controlled DMD clinical trials ever conducted, demonstrates Translarna’s benefits to patients and merits a full and fair review by the FDA, including an advisory committee meeting.
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