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.


In Canada, idebenone will be marketed under the trade name Catena and is expected to become available in late October. Under the conditions of the Canadian approval, Santhera agreed to provide additional data from the ongoing phase 3 trial in the United States so as to confirm efficacy of the treatment.

Also on July 24, the European Medicines Agency (EMEA) advised Santhera that it would not support marketing authorization for SNT-MC17/idebenone to treat patients with FA at this time. Santhera says the EMEA expressed a preference to await results of the phase 3 trials currently under way in both Europe and the United States.

Idebenone is not yet approved in the United States, where regulators also await the results of the phase 3 trial.

“We urge eligible FA patients to consider participation in such clinical trials,” said Jennifer Farmer, executive director of FARA. “The faster these trials obtain sufficient participants, the faster the drugs can be tested and submitted for approval.”

About Friedreich’s Ataxia
Friedreich’s ataxia, named after German physician Nikolaus Friedreich who described it in the 19th century, is a genetic disease that results in a deficiency of frataxin, a protein needed to regulate iron in cells. Patients experience progressive loss of coordination and strength; impairments in speech, hearing and vision; spinal curvature (scoliosis); diabetes; and cardiac muscle impairment.

About MDA
MDA is a voluntary health agency aimed at conquering more than 40 diseases through programs of worldwide research, comprehensive medical and community services, and far-reaching professional and public health education.

About FARA
FARA is a 501(c)(3) nonprofit charitable organization dedicated to accelerating research leading to treatments and a cure for Friedreich’s ataxia.

About Santhera
Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on the discovery, development and marketing of small-molecule pharmaceutical products for the treatment of severe neuromuscular diseases.