Neuraxpharm Pharmaceuticals S.L.

The European Medicines Agency’s human medicines committee again decided not to recommend the renewal of the conditional marketing authorization for PTC Therapeutics’ Duchenne muscular dystrophy drug Translarna. The treatment has gone through multiple reviews by the committee. Friday’s decision is the fourth time the EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended against the drug. The CHMP first decided against Translarna in September 2023, then confirmed that decision in January 2024, following a re-examination requested by PTC Therapeutics. In June, the committee reviewed the drug after the European Commission asked the committee to consider additional data — but the committee’s decision remained the same. PTC Therapeutics asked the committee again to reconsider in July, which led to this fourth decision. Since its initial authorization in 2014, PTC Therapeutics has carried out two more studies in an attempt to show the drug’s effectiveness. The first study failed to confirm the efficacy of the treatment, though the data suggested that a subgroup of patients — those with a progressive decline in their ability to walk — might see more efficacy. A second study did not reach statistical significance in that subgroup of patients. The drug will remain available while the EMA reviews CHMP’s recommendation. “CHMP again based its decision on the results of the primary analysis subpopulation of Study 041 instead of the totality of evidence for Translarna,” PTC Therapeutics CEO Matthew Klein said in a statement . Leerink Partners wrote Friday that they expect this to be “the final negative opinion,” adding that although PTC Therapeutics resubmitted an NDA to the FDA in late July, there have been no more updates in the US, and they “remain skeptical on the likelihood of US approval.” In addition to the Translarna decision, here’s a look at the medicines that CHMP recommended ahead of a potential thumbs-up from the European Commission: