3rd time's the charm? PTC will get another chance to keep Translarna's European marketing authorization

Four months after Europe's drug reviewers doubled down and recommended the withdrawal of PTC Therapeutics' Translarna for lack of efficacy, the drug's regulatory odyssey is taking yet another turn.

Monday, PTC Therapeutics said that the European Commission has spurned the recommendation by the Committee for Medicinal Products for Human Use (CHMP) to not renew the drug's marketing authorization.

The European Commission has sent the case back to the CHMP for another reevaluation, PTC said in a release. This development follows two prior CHMP reviews, which resulted in recommendations for withdrawal in September 2023 and January 2024.

In January, after a CHMP reexamination, PTC Therapeutics CEO Matthew Klein, M.D., acknowledged that the negative recommendation would "result in the withdrawal" of the drug in Europe. Still, even after a second review went against PTC's wishes, the company didn't stop voicing its protests about the possible market pull.

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

With the European Commission's recent decision, Translarna's market authorization remains intact as the new review plays out. The drug gained its European nod in 2017 to treat nonsense mutation Duchenne muscular dystrophy.

In another Monday disclosure, PTC said the European Medicines Agency has deemed "invalid" a Scientific Advisory Group (SAG) meeting for the drug that was held Sept. 5, 2023. The SAG's input "will not be considered by the CHMP in any future evaluation of Translarna," PTC said.

In conjunction with the evolving regulatory situation, PTC decided to pause its 2024 revenue guidance. Just last month, the company said it expected to generate $600 million to $680 million this year.

PTC's stock price climbed by about 21% to $40 before the U.S. markets opened Monday.

Even as the company fights to keep its Translarna market foothold in Europe, PTC in March said it plans to file for a potential FDA approval this year. The U.S. regulator rejected the drug in 2016 and 2017.