PTC Therapeutics (NASDAQ:PTCT) stock sank in Friday premarket trading after the European Medicines Agency (EMA) turned down full market authorization for Translarna, its key treatment for Duchenne muscular dystrophy.
Ahead of the opening bell, the company’s shares were down 29% at $26.56.
In a statement, the company said the EMA’s Committee for Medicinal Products for Human Use (CHMP) gave a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization for Translarna for treating nonsense mutation Duchenne muscular dystrophy (nmDMD).
Noting that the negative opinion also applies to the renewal of the existing conditional authorization, PTC said it plans to submit a request for re-examination per EMA guidelines.
"We are surprised and extremely disappointed by the CHMP decision, given the well-established and favorable safety and efficacy profile of Translarna," commented PTC Therapeutics (NASDAQ:PTCT) CEO Dr Matthew Klein.
"Of course, this decision is most devastating for the hundreds of boys and young men in Europe with nonsense mutation DMD, for whom no other approved therapies are available.”
The company said the CHMP’s decision was based on data from three placebo-controlled trials of the therapy. In the re-examination, it said it plans to focus on the clear and consistent evidence of benefit recorded in the overall population of 700 boys included in all 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 over 5.5 years, in supporting the benefits of long-term treatment.
The company said Translarna will remain on the market and available to patients with nmDMD until the re-examination process is completed. Based on CHMP procedural guidance, PTC said 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.
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