Saturday, September 16, 2023
PTC Therapeutics, Inc. has received an unfavorable decision from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) regarding the conversion of conditional marketing authorization to full marketing authorization for Translarna™ (ataluren) in the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). This negative opinion also extends to the renewal of the existing conditional authorization. PTC intends to request a re-examination following EMA guidelines. Translarna will continue to be available for nmDMD patients in the market during the re-examination process. The opinion following re-examination is expected in January 2024, with EC ratification within 67 days.
Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, expressed disappointment with the CHMP decision, emphasizing Translarna's established safety and efficacy profile. The conditional marketing authorization for Translarna in Europe dates back to 2014, with a renewal in 2017. A specific obligation for a third placebo-controlled trial, Study 041, was part of the conditional marketing authorization renewal. PTC presented Study 041 results, which showed nominally statistically significant results on key endpoints in the overall Intent-to-Treat population of 359 boys, although the primary analysis subgroup did not reach statistical significance.
Data from Study 041 and previous placebo-controlled trials, along with meta-analyses, were considered by the CHMP. Furthermore, analyses of the real-world STRIDE registry supported long-term Translarna therapy's benefits. Translarna demonstrated a favorable safety profile, with over 3,000 patients treated to date.
In the re-examination process, PTC plans to highlight the consistent evidence of benefit in the overall population of 700 boys across all studies (007, 020, and 041). They will address the decision to designate a different primary analysis subgroup in Study 041 and concerns regarding the robustness of the STRIDE data.