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PTC Therapeutics' DMD Drug to Remain Available in Europe as CHMP Reviews Opinion

NEW YORK – The European Commission has decided not to adopt the European Medicines Agency's Committee for Medicinal Products for Human Use's (CHMP) negative opinion on PTC Therapeutics' Duchenne muscular dystrophy therapy Translarna (ataluren), returning the opinion for further consideration, the biopharmaceutical company said Monday.

The CHMP in January issued a negative opinion for the annual renewal of the conditional marketing authorization of Translarna on the basis that its effectiveness had not been confirmed in clinical trials and that data from patient registries did not sufficiently link improvements in clinical outcomes to Translarna treatment. However, the EC has returned the opinion to the committee for reevaluation and asked it to further consider data from patient registries and real-world evidence in its revised opinion.

In the meantime, the drug's authorization will remain active in Europe and it will remain on the market.

"The maintenance of the current authorization of Translarna is a big win for boys and young men with nonsense mutation Duchenne muscular dystrophy in Europe," PTC CEO Matthew Klein said in a statement.

PTC's stock rose 21 percent to $40.12 per share at Monday's close, after closing at $33.10 on Friday. The stock price opened at $39.92 on Tuesday.

Translarna is a protein restoration therapy for patients with Duchenne muscular dystrophy caused by nonsense mutations and aims to enable cells to produce functional dystrophin by ignoring the early stop signal from the mutated dystrophin gene. The dystrophin protein is lacking or deficient in patients with Duchenne muscular dystrophy.

In September 2023, the CHMP recommended against converting Translarna's conditional marketing authorization into a full approval and against renewing the conditional marketing authorization for another year. In a study involving DMD patients whose ability to walk was progressively declining, there was no significant difference between a treatment and placebo arm when comparing distance covered in a six-minute walking test after 18 months on Translarna. In response to the decision, PTC requested the CHMP reexamine its decision; however, in January, the committee issued another negative opinion.

The EMA has said that input from scientific advisory group meetings on Translarna in September and January will not be considered in future evaluations, PTC said Monday.

Separately, in March, Warren, New Jersey-based PTC said it would resubmit a new drug application for Translarna to the US Food and Drug Administration, which also had previously said the company hadn't provided enough efficacy data. The company said it has factored in feedback from the US regulator in its resubmission.