PTC Pulls File for Duchenne Therapy on FDA Feedback

Why It Matters

The withdrawal eliminates the only US‑available nonsense‑mutation DMD therapy, leaving patients without a disease‑modifying option and pressuring PTC to rely on declining sales and new products. It also signals heightened regulatory scrutiny for rare‑disease drugs, influencing investor confidence and industry strategies.

Key Takeaways

• FDA rejects Translarna after decade-long US pursuit
• European approval withdrawn, conditional marketing not renewed
• PTC's US DMD sales fell to $50M, down $22M
• Company shifts focus to PKU drug Sephience
• Pipeline includes Huntington's and Friedreich’s ataxia candidates

Pulse Analysis

Translarna, known generically as ataluren, has been a focal point of the Duchenne muscular dystrophy (DMD) community for more than two decades. The drug targets nonsense‑mutation DMD, a subset of patients lacking functional dystrophin due to premature stop codons. After an initial refusal‑to‑file in 2016 and a subsequent FDA denial in 2017, PTC re‑entered the approval process in 2024, only to encounter the agency’s latest assessment that the clinical data fell short of the “substantial evidence” standard. The decision effectively closes the US pathway for a therapy that has already lost its conditional European marketing authorization.

The regulatory setback translates directly into PTC’s bottom line. Third‑quarter 2025 revenue from Translarna slipped to roughly $50 million, a $22 million decline from the prior year, while the company’s steroid‑based DMD product Emflaza also feels pressure from generic competition. In response, PTC is accelerating the launch of Sephience, a sepiapterin formulation for phenylketonuria, which generated $20 million in the same quarter. Parallel pipeline efforts—Novartis‑partnered votoplam for Huntington’s disease and vatiquinone for Friedreich’s ataxia—remain in early stages, but they now carry greater strategic weight for the firm’s growth.

The episode underscores a broader trend: regulators are demanding more robust efficacy signals for rare‑disease treatments, even when safety profiles are acceptable. For investors and biotech firms, the lesson is clear—early alignment with FDA expectations and transparent data interpretation are essential to avoid costly withdrawals. Patients and advocacy groups, meanwhile, may see a shift toward compassionate‑use programs or off‑label use while awaiting alternative therapies. PTC’s pivot illustrates how a single regulatory decision can reshape a company’s portfolio, market positioning, and long‑term R&D roadmap.

PTC pulls file for Duchenne therapy on FDA feedback

Phil Taylor · 13 February 2026

PTC Therapeutics has abandoned its latest attempt to secure FDA approval for Duchenne muscular dystrophy (DMD) treatment Translarna, after the FDA said it was unlikely to approve the drug based on the submitted data.

In a statement, PTC's chief executive, Matthew Klein, said the company had decided to pull the filing for Translarna (ataluren) as a treatment for nonsense‑mutation DMD after the FDA said it was “unlikely to meet the agency's threshold of substantial evidence of effectiveness.”

The latest setback looks like it could finally spell the end for Translarna in the US, after around a decade of attempts by PTC to bring it to market there. Its first attempt resulted in a refusal‑to‑file (RTF) letter in 2016, which was successfully appealed, but nevertheless followed by a rejection by the FDA in 2017.

After years of discussion and debate with the regulator, and various additional clinical trials, PTC resubmitted Translarna in 2024.

The drug was approved in Europe in 2014, but in 2024 the European Commission decided not to renew its conditional marketing authorisation after multiple reviews of its safety and efficacy.

“We have worked tirelessly for over two decades to develop a safe and effective therapy for boys and young men affected by nonsense‑mutation DMD in the US, and are disappointed that FDA approval cannot be achieved,” said Klein.

In a letter to the DMD community in the US, PTC said “differences in data interpretation” between the company and the FDA could not be resolved, adding that it would be deciding in the next few weeks what the implications would be on the supply of ataluren for those currently receiving therapy.

“We understand that this outcome is devastating for the [patient] community, the families who have participated in clinical trials for almost 20 years, and the families who continue to wait for a treatment option that addresses the underlying cause of nonsense‑mutation Duchenne muscular dystrophy.”

In its third‑quarter 2025 results, PTC reported sales of around $50 million for Translarna, down from $72 million a year earlier. The company's other DMD therapy – steroid‑based Emflaza (deflazacort) – is also in decline after losing patent protection.

PTC will now have to focus its attention on the rollout of Sephience (sepiapterin), its new treatment for people living with the rare disorder phenylketonuria (PKU), in the US and Europe. Third‑quarter 2025 sales were around $20 million.

Its pipeline also includes Novartis‑partnered votoplam for Huntington’s disease – which generated mid‑stage data last year – and vatiquinone for Friedreich’s ataxia, which was turned down by the FDA in 2025 with a request for more efficacy data.