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Update from PTC Therapeutics

December 13, 2017

Dear Duchenne Community,

As we enter the holiday season, I wanted to thank the community for all your support throughout the regulatory process for Translarna™ (ataluren) with the FDA. Your voice as passionate advocates has been instrumental in this process. As you know, we filed a Formal Dispute Resolution Request (FDRR) with the FDA a few weeks ago. This is a standard process that exists at the FDA which allows the appeal to be evaluated by the office above the Division of Neurology Products/Office of Drug Evaluation I, which is the Office of New Drugs. While the FDA guidance indicates that it is a 30-day process, it can take more time, which we anticipate will be the case. Depending on the content of the response we will determine our next steps. These processes unfortunately never go as quickly as we would like, and we will update the community once we have a resolution.

We truly appreciate all your support and dedication. Our goal remains to gain FDA approval so that we can provide Translarna to those in the US that may benefit. We are dedicated to the families and to reaching that goal.

We wish you all a joyful holiday season.


Stuart Peltz
CEO of PTC Therapeutics