PTC Therapeutics Says European Commission Returns Translarna Opinion To CHMP For Re-evaluation

(RTTNews) - PTC Therapeutics, Inc. (PTCT) announced Monday that the European Commission (EC) has decided not to adopt the CHMP's negative opinion of January 24, 2024 on the annual renewal of the conditional marketing authorization of Translarna (ataluren) and has returned the opinion to the CHMP for re-evaluation.

As a result, Translarna remains on the market and available for patients in Europe consistent with its current marketing authorization. The EC has asked the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in a revised opinion.

In addition, the European Medicines Agency (EMA) has informed PTC that the decision has been taken to consider the Scientific Advisory Group (SAG) meeting for Translarna held on September 5, 2023, and all the procedural steps that followed, as invalid.

Furthermore, the input from the SAG meeting held in September 2023 as well as the meeting held in January 2024 will not be considered by the CHMP in any future evaluation of Translarna.

Given the inability to accurately forecast the upside impact of the continued authorization of Translarna in Europe on 2024 revenue, PTC will pause 2024 total revenue guidance at this time.