European Commission Declines To Renew Marketing Authorization Of Translarna Duchenne Muscular Disorder

The European Commission (EC) adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) not to renew the authorization of PTC Therapeutics, Inc.'s PTCT Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy (nmDMD).

Also Read: PTC Therapeutics Reveals New Sepiapterin Data From Phase 3 Study In Patients With Inherited Disorder, FDA Decision Expected In July

While this action effectively removes the drug's conditional marketing authorization in the European Economic Area, the EC indicated that individual countries within the European Union could leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued use of Translarna.

"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 Matthew B. Klein, PTC Therapeutics CEO in a statement on Friday.

"We look forward to working on a country-by-country basis to provide commercial drug where possible."

In May 2024, PTC Therapeutics announced that the European Commission (EC) has decided not to adopt the CHMP's negative opinion on the annual renewal of Translarna's conditional marketing authorization (ataluren) and has returned the opinion to the CHMP for re-evaluation. 

Translarna's net product revenues were $339.9 million for the full year 2024, compared to $355.8 million for the full year 2023.

In 2024, PTC submitted four regulatory approval applications to the FDA, all of which have been accepted for review, including Translarna for nmDMD.

Price Action: PTCT stock is down 1.76% at $54.02 at the last check Friday.

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