PTC Therapeutics says EC not to renew market authorization for muscle disorder drug

Published: 2025-03-28 10:18:12 ET PTCT

(Adds background in paragraph 2, CEO statement in paragraph 4 and details on the drug in paragraphs 3, 5)

March 28 (Reuters) - PTC Therapeutics said on Friday its inherited progressive muscle-wasting disorder drug will no longer be available for sale in the EU as the European Commission has decided not to renew its authorization following a prolonged review.

The EC's decision backs the negative opinion on the drug given by the EU regulator's Committee for Medicinal Products for Human Use (CHMP) in 2023, which was then reaffirmed in January last year.

The drug, Translarna, had received conditional approval in the European Union in 2014 to treat children aged five years and older who can walk and are afflicted with Duchenne muscular dystrophy (DMD).

"We are of course disappointed that after this prolonged period of review the European Commission has decided to adopt the CHMP negative opinion on Translarna," said PTC CEO Matthew Klein.

Translarna is designed to boost production of a protein called dystrophin, the absence of which causes muscle weakening in patients with DMD. (Reporting by Christy Santhosh in Bengaluru; Editing by Shailesh Kuber)