PTC Therapeutics withdraws Translarna (ataluren) NDA for nonsense mutation Duchenne muscular dystrophy after FDA deems data insufficient. Explains ongoing expanded access supply, CEO statement, DMD background, and patient support options
Written By: Pharmacally Medical News Desk
PTC Therapeutics, Inc. has withdrawn its New Drug Application (NDA) resubmission for Translarna™ (ataluren), a protein restoration therapy for nonsense mutation Duchenne muscular dystrophy (nmDMD), following feedback from the U.S. Food and Drug Administration (FDA). The decision stems from irreconcilable differences in data interpretation across two decades of clinical studies.
FDA Feedback and PTC’s Response
In a letter to the U.S. Duchenne community, PTC revealed that recent FDA discussions confirmed “differences in data interpretation that cannot be successfully resolved to enable approval.” Despite evidence of safety and effectiveness from multiple trials, the agency views the data as insufficient for its approval threshold.
Resonating this in an official press release, Matthew B. Klein, M.D., PTC’s Chief Executive Officer, stated: “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.”
Dr. Klein added, “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.”
PTC described the move as “difficult,” recognizing its “devastating” impact on families involved in nearly 20 years of trials and those awaiting a therapy targeting the genetic root cause of nmDMD, which affects 10-15% of DMD cases.
About Translarna™ (Ataluren) and Duchenne Muscular Dystrophy
Translarna (ataluren) is designed to enable formation of a functioning protein in nonsense mutation disorders by promoting ribosomal read-through of premature stop codons. In nmDMD, this targets dystrophin deficiency.
Duchenne muscular dystrophy (DMD) is a rare, fatal genetic disorder primarily affecting males, causing progressive muscle weakness from early childhood. Without functional dystrophin essential for muscle stability patients often lose ambulation by age 10, followed by arm function loss, respiratory failure requiring ventilation, and cardiac complications leading to death in the mid-20s.
Translarna is approved in Europe and other regions for nmDMD patients aged 2-5 years via accelerated pathways but has faced repeated U.S. hurdles.
“We are humbled by the community’s commitment and courage and deeply appreciate the many years of steadfast support,” PTC noted, reaffirming its shared mission amid this setback.
This development highlights persistent challenges in rare disease approvals, where limited patient pools complicate substantial evidence requirements. PTC stock (PTCT) reacted in after-hours trading; monitor for broader implications in DMD pipelines, including gene therapies.
Next Steps for Patients and Community
PTC will assess ongoing supply for current U.S. patients over the coming weeks, promising updates soon. Families can reach the patient engagement team at patientengagement@ptcbio.com.
Reference
PTC Therapeutics Provides Regulatory Update on Translarna™, 12 February 2026, https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-provides-regulatory-update-translarnatm-2
Update to the U.S. Duchenne Community on the Ataluren (Translarna™) NDA Review, 12 February 2026, https://www.ptcbio.com/wp-content/uploads/sites/2/2026/02/PTC-Update-to-the-Duchenne-community-Feb-2026.pdf