Idebenone in Friedreich ataxia and Leber’s hereditary optic neuropathy: close mechanisms, similar therapy?

Manuel Schiff, Pierre Rustin. Brain (2016) aww085 DOI: 10.1093/brain/aww085 First published online: 19 April 2016

"Yet despite these studies, there is no clear consensus on the benefits of idebenone therapy for patients with Friedreich ataxia. Nevertheless, being essentially harmless idebenone has been, and is still, given to many patients with Friedreich ataxia all over the world."

However, for legal and/or marketing reasons, recent idebenone authorization for LHON resulted in some patients with Friedreich ataxia having difficulty obtaining it.

So far the occurrence of potential responders to idebenone in Friedreich ataxia has not been rigorously examined, but in view of the data obtained in LHON, defining a subgroup of therapy-responder patients appears a reasonable objective for next trials in this (and other) rare disease.

Idebenone in Friedreich ataxia and Leber’s hereditary optic neuropathy: close mechanisms, similar therapy?