Today PTC Therapeutics released an updated letter to the Duchenne community on their website with additional details following the company’s withdrawal of the ataluren New Drug Application (NDA). This update includes new information on a limited supply of therapy, which PTC anticipates to last up to approximately six months for those currently receiving treatment, as well as a dedicated email address for physicians with questions.
Based on interactions with the U.S. Food and Drug Administration (FDA), PTC announced in February 2026 that the company has formally withdrawn its resubmission of its NDA for ataluren and that the FDA accepted the withdrawal. As a result, ataluren is no longer under active review for approval to market a new drug product in the United States.
We recognize how difficult this moment is, and that this update brings additional uncertainty and frustration for many in our community. Please know that PPMD is here for you. If you need support, have questions, or want to talk, we encourage you to reach out to our care team directly: careteam@parentprojectmd.org or schedule a one-on-one meeting through our PPMD For You program.
PPMD is actively working to support the community in understanding the regulatory context of this development and what this means moving forward. We are planning an independent webinar in the coming days to walk through the regulatory landscape, answer questions, and provide additional clarity. Please stay tuned for additional details coming soon.
We will continue to share updates with the community as information becomes available. According to PTC, if your physician has questions, they can contact PTCActs@ptcbio.com.
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