PTC Therapeutics Provides Regulatory (FDA) Update on Translarna™

 PTC Therapeutics Provides Regulatory Update on Translarna™ 

February 12, 2026 

WARREN, N.J., Feb. 12, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that it has withdrawn the New Drug Application (NDA) resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following U.S. Food and Drug Administration (FDA) feedback on the application review. 

"FDA shared that based on its review to date, the data in the NDA submission are unlikely to meet the Agency's threshold of substantial evidence of effectiveness to support approval of Translarna. We have therefore made the decision to withdraw the NDA submission," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "We have worked tirelessly for over two decades to develop a safe and effective therapy for boys and young men affected by nonsense mutation DMD in the U.S. and are disappointed that FDA approval cannot be achieved." 

About Translarna™ (ataluren) Translarna (ataluren), is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne. 

Update to the U.S. Duchenne Community on the Ataluren (Translarna™) NDA Review: 

Recent discussions with the U.S. Food and Drug Administration (FDA) have made clear that there are differences in data interpretation that cannot be successfully resolved to enable approval of ataluren (Translarna™) for the treatment of nonsense mutation Duchenne muscular dystrophy in the U.S. Despite the evidence of safety and effectiveness demonstrated across several clinical studies, FDA has shared that they view the data as insufficient to meet their threshold for approval. Accordingly, PTC has made the difficult decision to withdraw the resubmission of the New Drug Application for ataluren. 

We understand that this outcome is devastating for the Duchenne muscular dystrophy community, the families who have participated in clinical trials for almost 20 years, and the families who continue to wait for a treatment option that addresses the underlying cause of nonsense mutation Duchenne muscular dystrophy. 

Over the coming weeks, we will be determining next steps regarding supply of ataluren for those currently receiving therapy. We will provide more information once we complete our assessment. If you have any questions, please feel free to reach out to our patient engagement team at patientengagement@ptcbio.com. 

We are humbled by the community's commitment and courage and deeply appreciate the many years of steadfast support in our shared mission. We are disappointed that, despite our best efforts, we have not been able to achieve FDA approval for ataluren.