What is ataluren and how does it work?
Ataluren is an investigational (experimental) drug that is the first potential new therapy designed to enable the formation of a functioning protein in a patient with a genetic disorder due to a nonsense mutation. Ataluren is taken orally and has the potential to treat the root cause of the disease by overriding the premature stop signal, so that a functional protein can be formed in a patient who has a disease due to a nonsense mutation. In other words, it seems to allow the body to overcome the genetic problem. It does not alter a patient’s genetic code or introduce genetic materials into the body. Ataluren is currently being tested in a Phase 2b trial in patients with nonsense mutation Duchenne/Becker muscular dystrophy and will soon be tested in a Phase 3 trial in nonsense mutation cystic fibrosis. These trials build on prior Phase 1 and 2 trials.
