The Muscular Dystrophy Association (MDA) and the Friedreich's ataxia Research Alliance (FARA) announced this week that, on July 24, the antioxidant compound idebenone received conditional market approval for the treatment of Friedreich's ataxia (FA) in Canada but failed to obtain such approval in Europe.
Idebenone, which is related to the natural substance coenzyme Q10, was developed by the Swiss pharmaceutical company Santhera as SNT-MC17.
The company is testing the compound, which is intended to improve cellular energy production and reduce oxidative stress, in FA, Duchenne muscular dystrophy (DMD) and Leber’s hereditary optic neuropathy.
In a phase 2 study in patients with FA, idebenone was generally well tolerated and associated with improvement in neurological function, as well as scores measuring activities of daily living.
A phase 3 trial in FA remains open.