European Authority Gives Thumbs Down To PTC Therapeutics' Duchenne Therapy; Analyst Says It Can Overturn The Challenge

Friday morning, the Committee for Medicinal Products for Human Use of the European Medicines Agency gave a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization of PTC Therapeutics Inc's PTCT Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy. 

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

PTC plans to submit a request for re-examination. Translarna will remain on the market and available until the re-examination process is completed. 

Following the re-examination process, the opinion would be expected to occur in January 2024, with the European Commission ratifying 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. 

PTC agreed to conduct a third placebo-controlled trial, Study 041, to renew the conditional marketing authorization. 

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.

William Blair considers the update to be unexpected and unfortunate; it's essential to note that PTC has previous experience in overturning the CHMP's stance on Translarna. 

In 2014, the CHMP initially issued a negative opinion regarding Translarna's conditional marketing authorization, and PTC successfully reversed that decision.

Moreover, in 2017, PTC effectively negotiated the renewal of Translarna's conditional marketing authorization, which included the commitment to conduct a long-term post-authorization trial.

Additionally, the analyst finds the change in rapporteur for the re-examination process and the positive data from all three studies, particularly when analyzing the 300-400m 6MWT baseline population as the primary group, to be supportive of the potential to overturn the CHMP's decision.

Price Action: PTCT shares are down 28.70% at $26.66 on the last check Friday.