On October 25, PTC Therapeutics shared the news that the FDA had issued a Complete Response Letter regarding their application for ataluren, noting that FDA had stated that “an additional adequate and well-controlled clinical trial(s) will be necessary at a minimum to provide substantial evidence of effectiveness.” On that day, PTC also announced that they planned to file a formal dispute resolution and – as of this week – have done so.
PPMD has been working to understand the formal dispute resolution process and what opportunities may exist for our community to be of support.
We understand from the Guidance for FDA Formal Dispute Resolution that this is a formal regulatory process that will transpire between PTC and the FDA. While we are not privy to the content of the confidential documents that have been exchanged between the two entities, we have consulted with our regulatory advisors, familiarized ourselves with the process and the Guidance for FDA Formal Dispute Resolution, and reached out to the FDA about whether there is any way that we can be of support. We will continue to communicate anything we learn as soon as we learn it.
Thank you to all the families and Duchenne experts who raised their voices at the Advisory Committee meeting and through written testimony. We appreciate the effort of families who are continuing to raise awareness through social media with their personal stories. We also thank all those boys, men, and families who are continuing to support PTC through their continued participation in the on-going extension study.
Our Duchenne community is remarkable.