PTC Therapeutics Announces European Commission Returns Translarna Opinion to CHMP For Re-evaluation

- European Commission decides against adoption of negative opinion -
- Translarna authorization remains active in Europe -
- 2024 revenue guidance paused -

WARREN, N.J., May 20, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has decided not to adopt the CHMP's negative opinion of Jan. 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.

"The maintenance of the current authorization of Translarna is a big win for boys and young men with nonsense mutation Duchenne muscular dystrophy in Europe," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. "The efficacy and safety demonstrated in clinical trials and the long-term STRIDE registry support that Translarna fills an otherwise unmet need for nmDMD patients. We look forward to working collaboratively with CHMP on next steps once they are defined."

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 Sept. 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. Guidance will be updated at a future date.

About Translarna (ataluren)
Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne. Translarna, the tradename of ataluren, is licensed in multiple countries for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged 2 years and older. Ataluren is an investigational new drug in the United States.