What is ataluren (formerly called PTC124®)?
Ataluren is an investigational (experimental) drug, which means it is being tested as a potential treatment and it has not yet been approved for sale by the U.S. Food and Drug Administration (FDA), the European Medicines Evaluation Agency (EMEA) or any other regulatory authority. Ataluren has also been known by the name of PTC124®. Ataluren is the first potential 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. Ataluren does not alter a patient’s genetic code or introduce genetic materials into the body. In hemophilia A and B, it may allow the body to produce increased amounts of the Factor VIII and IX pro
