RARE Daily

CHMP Calls for Pulling PTC’s DMD Drug Translarna off the Market Following Re-examination

January 26, 2024

Rare Daily Staff

The E.U.’s Committee for Medicinal Products for Human Use issued a negative opinion following the re-examination procedure for the conditional marketing authorization of PTC Therapeutics’ Translarna after finding that the data do not support its safety and efficacy.

The European Union granted conditional approval to Translarna in 2014 to treat ambulatory children aged five years and older with Duchenne muscular dystrophy expressing nonsense mutations. The European Commission has 67 days to adopt CHMP’s negative opinion, which will result in ataluren being taken off the European market.

“The CHMP’s decision, which is against the stated wishes of the patient community and expert physicians, will be devastating for children and young men in Europe for whom no other disease-modifying therapies are available,” said Matthew Klein, CEO of PTC Therapeutics.

Primarily affecting males, 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.

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