PTC Therapeutics withdrew its New Drug Application (NDA) resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy (nmDMD) on February 12, 2026, following FDA feedback that the data were unlikely to meet the threshold of substantial evidence of effectiveness.123
CEO Matthew B. Klein expressed disappointment, noting over two decades of development efforts but that FDA approval cannot be achieved.134
Translarna is a protein restoration therapy aimed at enabling production of functional dystrophin in nmDMD, a rare progressive genetic disorder causing muscle weakness and early death.13
The withdrawal represents a significant regulatory setback for Translarna's US approval, with PTC assessing next steps for ongoing therapy supply.2
PTC stock fell 4-5% in after-hours trading following the announcement.45
Sources:
1. https://www.stocktitan.net/sec-filings/PTCT/8-k-ptc-therapeutics-inc-reports-material-event-a3bfba202f19.html
2. https://www.ptcbio.com/wp-content/uploads/sites/2/2026/02/PTC-Update-to-the-Duchenne-community-Feb-2026.pdf
3. https://www.prnewswire.com/news-releases/ptc-therapeutics-provides-regulatory-update-on-translarna-302686879.html
4. https://www.investing.com/news/stock-market-news/ptc-therapeutics-stock-falls-after-withdrawing-translarna-drug-application-93CH-4504118
5. https://www.morningstar.com/news/dow-jones/2026021217907/ptc-therapeutics-pulls-fda-application-for-muscular-dystrophy-treatment