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Monday, PTC Therapeutics Inc (NASDAQ:PTCT) announced that the European Commission (EC) has decided not to adopt the CHMP’s negative opinion on the annual renewal of the conditional marketing authorization of Translarna (ataluren) and has returned the opinion to the CHMP for re-evaluation.
As a result, Translarna remains on the market and available for patients in Europe, consistent with its current marketing authorization.
The EC has asked the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in a revised opinion.
In addition, the European Medicines Agency (EMA) has informed PTC that it has decided to consider the Scientific Advisory Group (SAG) meeting for Translarna held on September 5, 2023, and all the procedural steps that followed as invalid.
Furthermore, the input from the SAG meeting held in September 2023 and the meeting held in January 2024 will not be considered by the CHMP in any future evaluation of Translarna.
In January, the Committee for Medicinal Products for Human Use (CHMP) of the EMA issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarna.
In September 2023, the CHMP gave a negative opinion on converting the conditional marketing authorization to full marketing authorization of Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy.
Given the inability to accurately forecast the upside impact of the continued authorization of Translarna in Europe on 2024 revenue, PTC will pause 2024 total revenue guidance of $600 million-$680 million.
Translarna, the tradename of ataluren, is licensed in multiple countries for nonsense mutation Duchenne muscular dystrophy in ambulatory patients (who can walk) aged two years and older.
PTC plans to resubmit the U.S. marketing application for Translarna for nmDMD in mid-2024.
Translarna net product revenues were $103.6 million for the first quarter of 2024.
Price Action: PTCT shares are up 18.3% at $39.16 at last check Monday.
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Posted In: PTCT
