英文药名：Raxone（idebenone film-coated tablet）
通用名：idebenone film-coated tablet
Raxone®(idebenone)150mg film-coated tablet
The European Commission has granted marketing authorization for Raxone (idebenone) for the treatment of visual impairment in adolescent and adult patients with Leber's Hereditary Optic Neuropathy (LHON).
This rare condition is an inherited mitochondrial disease that can lead to rapid, profound and permanent blindness in otherwise healthy patients. Typically it occurs in young adults but it can occur at any age. The rapid vision loss is believed to be the result of mitochondrial mutations that results a loss of function among several NADH-ubiquinone oxidoreductase chains.
Idebenone is a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1).
Efficacy of Idebenone in Leber's Hereditary Optic Neuropathy
In 2011, Klopstock et al1 reported that the primary endpoint (best recovery in visual acuity) was not achieved in their 24-week multicenter, placebo-controlled, randomized, double-blind study involving 85 patients with LHON. However, numerous secondary measures did reach statistical significance (changes in visual acuity in best eye; changes in visual acuity for both eyes in each patient).
A follow-up study in 60 of the 85 patients, showed a similar pattern of visual improvement that was not statistically significant.2
In a statement3, lead investigator of those studies— Thomas Klopstock, MD, Professor for Neurology at the University of Munich—said, "Raxone represents a major breakthrough in mitochondrial disease treatment, and its approval paves the way for patients with LHON to be treated and to achieve a meaningful improvement of their visual acuity."
Roxane (idebenone) treatment (900 mg daily given as 2 tablets three times a day with food) should be initiated and supervised by a physician with experience in LHON
Approximately 8000 people in Europe have LHON.
Also Being Studied for Duchenne Muscular Dystrophy and Multiple Sclerosis
In the United States, idebenone has orphan designation for LHON but it is not known when an NDA will be filed. The drug is also being investigated for treatment of Duchenne muscular dystrophy (phase 3 study) and multiple sclerosis (phase 2 study).