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

In August 2014 Translarna received marketing authorization in the European Union for the treatment of nonsense mutation Duchenne in ambulatory patients aged five years and older, representing the first-ever treatment approved for the underlying cause of the disease.

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.

A Phase 3 study is currently actively recruiting around the world. Two trials to study dystrophin levels in nonsense mutation patients (both Translarna-treated and untreated) also began recruiting in 2019.

Sponsor

This program is sponsored by PTC Therapeutics.

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