PTC Therapeutics Provides Regulatory Update and Withdraws Its New Drug Application for Translarna™

We are disappointed to report that PTC Therapeutics has withdrawn its New Drug Application for Translarna™ (ataluren) for the treatment of nonsense mutation DMD after feedback from the FDA that the data are insufficient to meet the standard for approval. The company is expected to inform the community in the coming weeks of next steps for those currently receiving Ataluren therapy.   

Our hearts are especially with the families who have participated in this clinical program for years—those who committed their time and endured countless visits in hopes of moving the science forward. We recognize how heavy this moment may feel.

We are deeply grateful to these families and to the team at PTC who dedicated years of work to advancing this research. Progress in Duchenne is only possible because of that commitment.

CureDuchenne stands with every family impacted, and we remain committed to accelerating promising research and expanding options so that no one with Duchenne is left behind.

Read the full press release: https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-provides-regulatory-update-translarnatm-2

Read their community letter: https://www.ptcbio.com/wp-content/uploads/sites/2/2026/02/PTC-Update-to-the-Duchenne-community-Feb-2026.pdf