Duchenne can be caused by a number of genetic mutations (changes). Ataluren (Translarna®) is for use in patients with “nonsense mutations” in the dystrophin gene, which prematurely stop the production of a normal dystrophin protein and lead to a shortened and nonfunctional dystrophin protein. Translarna works in these patients by enabling the protein-making apparatus in cells to move past the nonsense mutation, allowing the cells to produce a functional dystrophin protein.

Status

On September 15, 2023 EMA recommended non-renewal of authorization of Duchenne muscular dystrophy medicine Translarna, PTC has appealed this decision. PTC is expanding commercial access to Translarna across Europe, the Middle East, Latin America, and Asia Pacific.

PTC has engaged in dialogue with the US Food and Drug Administration regarding a path forward to bring Translarna to patients in the US.

Sponsor

This program is sponsored by PTC Therapeutics.

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