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 call comes after the US Food and Drug Administration (FDA) voiced concerns over the drug’s efficacy in nonsense mutation DMD. According to PTC’s CEO, Matthew Klein, the regulator shared that based on its review thus far, the data used to support the application is “unlikely to meet the agency’s threshold of substantial evidence of effectiveness” to support Translarna’s approval.
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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 Pharmaceutical Technology’s request when asked if it plans to re-file for the drug’s approval with the FDA.
The FDA previously refused to review the dystrophin protein restoration therapy in 2016, citing the incomplete nature of its associated NDA as the primary reason.
On PTC’s second attempt to commercialise the drug in 2017, which followed a successful appeal of the FDA’s 2016 decision, the drug was once again rejected. This time, the decision was on the grounds that at least one additional study would be necessary to prove the drug’s efficacy in DMD.
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.
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By GlobalDataWhile 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, following the review by the European Commission (EC) in late 2023. Now, the drug is only available to countries choosing to leverage Articles 117(3) and 5(1) of the EU Directive 2001/83, according to a 2025 statement from PTC.
However, the drug remains approved for use in the UK. The drug is also available through the National Health Service (NHS).
GlobalData, parent company of Pharmaceutical Technology, 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, as Capricor Therapeutics received a complete response letter (CRL) for its cell therapy, deramiocel, back in September 2025. In January 2026, the company issued an update, stating that it is currently compiling a clinical study report and supporting data in a bid to resubmit its application for deramiocel in DMD.
Meanwhile, Sarepta’s approved DMD gene therapy, Elevidys (delandistrogene moxeparvovec), was also temporarily pulled from the US market following two non-ambulatory patient deaths after treatment. The FDA later reinstated Elevidys on the market following a review.
