PTC Therapeutics Pulls Translarna NDA, Faces Sales Forecast Drop from $356M to $73M
PTC Therapeutics has withdrawn its resubmitted NDA for Translarna after the FDA deemed its efficacy data insufficient to meet the substantial evidence threshold, marking the third US rejection since 2016. GlobalData forecasts Translarna’s sales will plunge from $356 million in 2023 to $73 million by 2031.
1. Withdrawal of NDA
On February 16, 2026, PTC Therapeutics withdrew its resubmitted NDA for Translarna, its therapy targeting nonsense mutation Duchenne muscular dystrophy in the US. This marks the third unsuccessful bid to secure FDA approval since the initial 2016 application.
2. FDA Feedback and Historical Rejections
The FDA determined the current efficacy data for Translarna was unlikely to achieve substantial evidence of effectiveness, prompting the withdrawal. Prior attempts in 2016 and 2017 were rejected due to incomplete data and the need for an additional efficacy study, respectively.
3. Sales Forecast and Market Outlook
GlobalData forecasts Translarna’s global sales will fall from a $356 million peak in 2023 to $73 million by 2031, reflecting diminished US prospects. While the drug lost EMA renewal in 2024, it remains approved for use in the UK and through select EU directives.