PTC's re-examination bid for DMD drug Translarna falls short in Europe

A decade after Translarna's initial marketing authorization in Europe, it looks like the drug's run in the region is coming to an end.

Thursday, PTC Therapeutics said a European Medicines Agency (EMA) committee again issued a negative opinion on renewing the Duchenne muscular dystrophy drug's authorization. The latest decision comes after a negative recommendation in September and a re-examination process.

After the re-examination, the Committee for Medicinal Products for Human Use (CHMP)'s decision is unchanged, PTC Therapeutics said in a brief release.

The decision goes "against the stated wishes of the patient community and expert physicians" and "will be devastating for children and young men in Europe for whom no other disease-modifying therapies are available," CEO Matthew Klein said in a statement. The CHMP's decision "will result in the withdrawal" of Translarna in Europe, Klein added.

PTC's Translarna won its initial conditional marketing authorization in Europe in 2014. European regulators then renewed the drug's authorization in 2017.

As part of the latest renewal process, the CHMP looked at Translarna’s data from the placebo-controlled Study 041, which PTC agreed to run in order to secure the renewal.

In the study, the drug failed to meet statistical significance on its primary endpoint in a selected patient subgroup, but it did turn in "nominally statistically significant results on several key endpoints," PTC said at the time of the September decision. The study's primary endpoint looked at patients' changes from baseline in a 6-minute walking distance test.

In September, the EMA said the committee found that the "results failed to confirm Translarna’s efficacy."

"These results were considered particularly relevant since the study included a population of patients expected to be most sensitive to the medicine," the agency said at the time.

In the wake of the latest decision, the European Commission has about 2 months to ratify the decision.

After the CHMP's September decision, PTC tripled the number of people who would be affected by a round of layoffs. Last May, the company said it would trim its headcount by about 8%. Weeks after the CHMP's decision, the company said it planned to trim around 25% of its workforce.

PTC's stock price slipped around 11% after the news on Thursday.