PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today 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.
- 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 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. “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 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. Guidance will be updated at a future date. About Translarna™ (ataluren) About Duchenne Muscular Dystrophy (Duchenne) About PTC Therapeutics, Inc. For More Information: Investors: Media: Forward-Looking Statement PTC’s actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC’s products or product candidates that PTC commercializes or may commercialize in the future; PTC’s ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia, the European Economic Area (EEA) and other regions; PTC’s ability to use the clinical data from its international drug registry study and real-world evidence concerning Translarna’s benefits to support a continued marketing authorization for Translarna for the treatment of nmDMD in the EEA; whether investigators agree with PTC’s interpretation of the results of clinical trials and the totality of clinical data from PTC’s trials in Translarna; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC’s products and product candidates; PTC’s scientific approach and general development progress; and the factors discussed in the “Risk Factors” section of PTC’s most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC’s other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Translarna. The forward-looking statements contained herein represent PTC’s views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. Acronyms: nmDMD: nonsense mutation Duchenne muscular dystrophy View original content:https://www.prnewswire.com/news-releases/ptc-therapeutics-announces-european-commission-returns-translarna-opinion-to-chmp-for-re-evaluation-302149749.html SOURCE PTC Therapeutics, Inc. | ||
Company Codes: NASDAQ-NMS:PTCT |