PTC's Translarna Gets CHMP's Negative Opinion For Conversion To Full Marketing Authorization

(RTTNews) - PTC Therapeutics, Inc. (PTCT) announced Friday that the European Medicines Agency's Committee for Medicinal Products for Human Use or CHMP has given a negative opinion on Translarna (ataluren) for conversion to full authorization.

The company was seeking the conversion of the conditional marketing authorization to full marketing authorization of Translarna to treat nonsense mutation Duchenne muscular dystrophy or nmDMD.

Duchenne, which primarily affects males, is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20's due to heart and respiratory failure.

The negative opinion also applies to the renewal of the existing conditional authorization.

Translarna received conditional marketing authorization in Europe in 2014 based on the results of Study 007. The conditional authorization was renewed in 2017, and as part of the renewal, PTC agreed to a specific obligation to conduct a third placebo-controlled trial, Study 041.

The trial results showed 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.

PTC said it plans to submit a request for re-examination to the CHMP per EMA guidelines to reverse this opinion, as it has done previously in the regulatory history of Translarna in Europe.

Translarna will remain on the market and available to patients with nmDMD until the re-examination process is completed.

The CHMP opinion following the re-examination process is expected to occur in January 2024, with EC ratification of the opinion within the following 67 days.

Matthew Klein, Chief Executive Officer, PTC, said, "We are surprised and extremely disappointed by the CHMP decision, given the well-established and favorable safety and efficacy profile of Translarna. 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."

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