CHMP negative decision on the conversion of the conditional marketing authorization to full marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchennemuscular dystrophy

The negative opinion also applies to the renewal of the existing conditional authorization. PTC plans to submit a request for re-examination per EMA guidelines. Translarna will remain on the market and available to patients with nmDMD until the re-examination process is completed. Based on CHMP procedural guidance, the opinion following the re-examination process would be expected to occur in January 2024, with EC ratification of the opinion within the following 67 days.

Translarna received conditional marketing authorization in Europe in 2014 based on the results of Study 007, and the conditional authorization was renewed in 2017. As part of the renewal of the conditional marketing authorization, PTC agreed to a specific obligation to conduct a third placebo controlled trial, Study 041. PTC shared the results of Study 041, which included nominally statistically significant results on several key endpoints in the overall enrolled Intent-to-Treat population of 359 boys even though it did not meet statistical significance in the primary analysis subgroup. The data from Study 041, as well as data from the two previous placebo-controlled trials—Studies 007 and 020 — formed the basis of the CHMPs review and decision. Additional supportive evidence of efficacy included robust meta-analyses demonstrating highly statistically significant benefit on several key disease endpoints capturing different aspects of DMD, including the Six-Minute Walk Distance test (6MWD), North Star Ambulatory Assessment, and Timed Function Tests. In addition, analyses of the real-world STRIDE registry, which includes 300 boys with an average treatment duration of over 5.5 years, demonstrating that long-term Translarna therapy delays the time to loss of ambulation by 3.5 years were included as part ofthe data package to confirm long-term meaningful treatment benefit. In addition, Translarna has shown a favorable safety profile, with over 3,000 patients treated to date.