PTC Therapeutics has chosen to withdraw its resubmitted new drug application (NDA) for Duchenne muscular dystrophy (DMD) therapy, Translarna (ataluren), marking another significant setback in the drug’s US development programme. This verdict could spell the end for Translarna’s decades-long and tumultuous US DMD development journey.
This call comes after the US Food and Drug Administration (FDA) voiced concerns over the drug’s efficacy in nonsense mutation DMD.
For this reason, the rare disease specialist has chosen to pull the NDA submitted to the FDA for Translarna in DMD, marking the third unsuccessful attempt by PTC to secure Translarna’s approval in the US. The company did not immediately respond to requests regarding future plans to re-file for the drug’s approval.
The FDA previously refused to review the dystrophin protein restoration therapy in 2016, citing the incomplete nature of its associated NDA. On PTC’s second attempt in 2017, the drug was rejected on the grounds that at least one additional study would be necessary to prove efficacy.
Translarna’s future in DMD market under question
Alongside its troubles in getting the US regulatory greenlight, Translarna has also been subject to regulatory woes in the European market. While the drug first gained conditional marketing authorisation from the European Medicines Agency (EMA) in 2014, the agency later issued a negative opinion on renewing Translarna’s approval in 2024. Now, the drug is only available to countries choosing to leverage specific articles of the EU Directive, though it remains approved for use in the UK via the NHS.
Market Impact: GlobalData forecasts that Translarna will generate $73m in global sales during 2031 – down significantly from its $356m 2023 sales peak.
Translarna is not the only DMD drug to receive a negative opinion from the FDA in recent months:
- Capricor Therapeutics received a complete response letter (CRL) for its cell therapy, deramiocel, in September 2025.
- Sarepta’s Elevidys (delandistrogene moxeparvovec) was temporarily pulled from the US market following safety concerns but was later reinstated following review.
