The European Commission made the rare decision Monday not to adopt the negative opinion of the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) on the renewal of Translarna’s conditional authorization.
The EC sent the opinion back to the CHMP for further evaluation, allowing the Duchenne muscular dystrophy therapeutic to remain on the market for now. PTC Therapeutics wrote that the EC “asked the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in a revised opinion.”
The company added that the September 2023 Scientific Advisory Group meeting for Translarna is considered invalid, and both the September and January 2024 meetings won’t be considered by CHMP in its re-evaluation of the drug.
In September last year, the CHMP recommended against giving a full marketing authorization to the drug, approved for nonsense mutation Duchenne muscular dystrophy, and also recommended against its conditional authorization. It’s had conditional authorization since 2014, and also received its first negative CHMP opinion that same year.
The drug brought in $355.8 million in 2023 and $103.6 million in the first quarter of 2024, with TD Cowen analysts writing that the EU accounts for about 50% of the drug’s worldwide sales.
It is yet to score an approval in the US, though PTC wrote in its latest financial report that based on agency feedback, it plans to resubmit its NDA for Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy in the middle of this year.
PTC said that it would hold off on its 2024 total revenue guidance because of the “inability to accurately forecast the upside impact of the continued authorization of Translarna in Europe.”
TD Cowen analysts wrote in a note that the EC’s decision was “unconventional” and that this “outcome is surprising, given that the EC is not a scientific body, and the vast majority of CHMP decisions are ratified.”
“Given the relative rarity of cases where the EC has not upheld the CHMP opinion, the eventual outcome is difficult to predict,” TD Cowen analysts added. “Given Translarna’s solid financial performance, any additional time the therapy remains on the market will be positive for PTC…We have noted that PTC’s performance this year will largely depend on regulatory flexibility across the EU and US, and today’s news reinforces that.”
PTC’s stock $PTCT was up nearly 20% Monday following the news.