PTC Duchenne drug approval set to be revoked in Europe

European authorities are set to rescind the approval of a Duchenne muscular dystrophy drug that’s been on the market there for nearly a decade following evidence the medicine, Translarna, doesn’t appear to help people with the condition.

In a statement Friday, the European Medicines Agency’s drugs committee recommended for the second time against renewing the drug’s clearance. The European Commission has 67 days to formally ratify the opinion in a legally binding decision for EU member states. If it does, Translarna would be removed from the European market, where it’s been available since 2014.

The recommendation is the latest development in Translarna’s controversial regulatory history. The drug, known scientifically as ataluren, is designed to block the effects of so-called nonsense mutations, or genetic alterations that stop the production of certain proteins. PTC developed the medicine for subsets of patients with cystic fibrosis and Duchenne, but has struggled for years to prove its benefit.

The drug failed in testing in cystic fibrosis and has been rejected multiple times by U.S. regulators for Duchenne. It was granted a “conditional” authorization by the EMA in 2014 despite mixed evidence in clinical testing. At the time, the regulator said the drug showed “some evidence of efficacy” in Duchenne, but noted the company would have to provide “comprehensive” data from another trial to uphold the approval.

Translarna was subsequently approved in other countries, including Russia and Brazil, and became PTC’s most lucrative wholly-owned product. Last year, it generated $355 million in sales.

Yet in 2022, Translarna failed the main goal of the study meant to confirm its benefits. Though PTC claimed other study findings proved the drug helps treat Duchenne, the EMA last year declined to convert its conditional approval to a standard clearance and sought to remove it from market. PTC appealed and sought a re-examination, but got the same result Friday.

In a written explanation, the EMA’s committee said PTC didn’t prove Translarna could slow a decline in walking ability among patients the company expected to benefit the most. Translarna has a “very small effect” on the production of a muscle-protecting protein, dystrophin, that Duchenne patients lack, while data from patient registries failed to provide conclusive evidence, the committee said.

The regulator “considered all the data available and the views of experts, patients and third parties” and “acknowledged the high unmet medical need for an effective treatment for patients with Duchenne.” But it concluded testing has not confirmed Translarna’s benefit.

In a statement, PTC CEO Matthew Klein argued the company’s data show Translarna is “safe and effective,” and cited support from the patient community and physicians.

PTC hasn’t given up seeking a clearance of Translarna in the U.S., meanwhile. It plans to meet with the Food and Drug Administration this quarter to discuss a path for an approval application.

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