RARE Daily

European Commission Returns Translarna Opinion to CHMP For Re-evaluation

May 20, 2024

Rare Daily Staff

The European Commission has decided not to adopt the Committee for Medicinal Products’ negative opinion of January 24, 2024 on the annual renewal of the conditional marketing authorization of PTC Therapeutic’s Duchenne muscular dystrophy therapy Translarna and has returned the opinion to the CHMP for re-evaluation.

The CMHP in January issued a negative opinion after finding that the data did not support the safety and efficacy of Translarna. The EU originally granted conditional approval for Translarna in 2014 for children five years and older with Duchenne muscular dystrophy expressing nonsense mutations.

As a result of the European Commission’s decision, 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.

Duchenne 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. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation) as early as 10 years old, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and 20s.

Translarna, discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne. Translarna, the tradename of ataluren, is licensed in multiple countries for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged 2 years and older. Ataluren is an investigational new drug in the United States.

In addition, the European Medicines Agency has informed PTC that the decision has been taken to consider the Scientific Advisory Group meeting for Translarna held on September 5, 2023, and all the procedural steps that followed, as invalid. Furthermore, the input from the Scientific Advisory Group held in September 2023 as well as the meeting held in January 2024 will not be considered by the CHMP in any future evaluation of Translarna.

PTC said it will pause 2024 total revenue guidance at this time because of the inability to accurately forecast the upside impact of the continued authorization of Translarna in Europe on 2024 revenue. It will update guidance at a future date.

“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,” said Matthew Klein, CEO of PTC Therapeutics. “The efficacy and safety demonstrated in clinical trials and the long-term STRIDE registry support that Translarna fills an otherwise unmet need for [nonsense mutation] DMD patients. We look forward to working collaboratively with CHMP on next steps once they are defined.”

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