PTC Therapeutics withdraws Translarna NDA after FDA cites insufficient efficacy data
PTC Therapeutics has withdrawn its NDA for Duchenne muscular dystrophy therapy Translarna after the FDA deemed efficacy data unlikely to meet substantial evidence thresholds, marking the third failed US approval attempt. GlobalData projects Translarna’s global sales to fall to $73 million by 2031 from $356 million in 2023.
1. Third US NDA Withdrawal
PTC Therapeutics elected to withdraw its resubmitted NDA for Translarna in Duchenne muscular dystrophy after the FDA determined that existing efficacy data was unlikely to meet the agency’s substantial evidence standard, marking the third unsuccessful US approval attempt since 2016.
2. EU and UK Regulatory Status
Translarna lost European Medicines Agency renewal in 2024 and now relies on Articles 117(3) and 5(1) of the EU Directive 2001/83 for access in select EU countries, while retaining approval and reimbursement under the UK National Health Service.
3. Declining Sales Forecast
GlobalData forecasts Translarna’s global sales will slump to $73 million by 2031, down from $356 million at its 2023 peak, reflecting mounting regulatory challenges and the absence of US market approval.