PTC Therapeutics provided an update to the Duchenne community with additional details following the withdrawal of the ataluren New Drug Application (NDA).
The update notes that a limited remaining supply of Ataluren is still available, anticipated to last up to six months for individuals currently receiving treatment, and provides details for treating physicians to continue to get access to the drug. Following interactions with the FDA, PTC confirmed in February 2026 that it formally withdrew its NDA resubmission for ataluren, which the FDA has accepted. Ataluren is no longer under active review in the United States.
We know this news impacts families in our community. CureDuchenne remains committed to advancing research and supporting every individual and family navigating Duchenne.
Read the Community Letter:
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