PTC Therapeutics ( PTCT Quick Quote PTCT - Free Report) announced that it received a negative opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for converting conditional marketing of Translarna (ataluren) to full marketing authorization. Shares of the company were down 30% on Sep 18, following the news.
Furthermore, the negative opinion also applies to the renewal of the existing conditional authorization of Translarna, which had received conditional marketing authorization in Europe in 2014 to treat nonsense mutation Duchenne muscular dystrophy (nmDMD). This authorization was subsequently renewed in 2017.
In response to the negative opinion, PTCT plans to request a formal re-examination procedure of the regulatory application per the EMA guidelines. The re-examination process is expected to occur in January 2024. Translarna will continue to remain on the market and available to patients with nmDMD until the re-examination process is completed.
Shares of PTC Therapeutics have risen 20.8% year to date against the
industry’s 14.0% decline. Image Source: Zacks Investment Research
The CHMP’s decision was based on data from three placebo-controlled studies (Study-007, Study-041 and Study-020) of the Translarna therapy. In addition to the clinical study data, the CHMP considered additional supportive evidence of the drug's efficacy. This included the results of robust meta-analyses, which are comprehensive statistical analyses that showed statistically significant benefits.
The analysis of the real-world STRIDE registry data was also considered by CHMP. The data from this registry indicated that long-term Translarna therapy could delay the time it takes for patients to lose their ability to walk.
In the re-examination process, PTCT intends to focus on the clear and consistent evidence of benefit recorded in the overall population of 700 boys included in all three studies. It will also address concerns raised regarding the robustness of data from a STRIDE registry, which includes 300 boys with an average treatment duration of more than 5.5 years, supporting the benefits of long-term treatment.
Zacks Rank & Stocks to Consider
PTC Therapeutics currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the same industry are
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