Date: 2017-05-02
Type of information: Distribution agreement
Compound: Raxone® (idebenone)
Company: Santhera Pharmaceuticals (Switzerland) Pharmathen (Greece)
Therapeutic area: Rare diseases - Genetic diseases - Ophtalmological diseases
Type agreement: distribution - supply
Action mechanism:
- benzoquinone analog/quinone. Raxone® (idebenone), a synthetic short-chain benzoquinone and a cofactor for the enzyme NAD(P)H:quinone oxidoreductase (NQO1), is capable of transferring electrons directly onto complex III of the mitochondrial electron transport chain, thereby circumventing the complex I defect and restoring cellular energy levels in retinal ganglion cells and promoting recovery of visual acuity. Nerve and muscle cells, including heart muscle cells, are particularly energy-demanding and are, therefore, more prone to rapid cell damage or death due to mitochondrial dysfunction. Through preserving mitochondrial function and protecting cells from oxidative stress, it is believed that Raxone®/Catena® can prevent cell damage and increase the production of energy within impaired nerve and muscle tissue in Friedreich's Ataxia and Duchenne patients.
- Raxone® is the first treatment option for LHON and the first approved therapy for a mitochondrial disease.
Disease: Leber's hereditary optic neuropathy (LHON)
Details:
- • On May 2, 2017, Santhera Pharmaceuticals announced that it has signed an agreement with Pharmathen to market Raxone® for the treatment of Leber's hereditary optic neuropathy (LHON) in Greece and Cyprus. Raxone® is authorized at a daily dose of 900 mg (given as 2 tablets three times a day with food), for the treatment of visual impairment in adolescent and adult patients with LHON. Treatment should be initiated and supervised by a physician with experience in LHON. Efficacy data come from Santhera's randomized, placebo-controlled RHODOS trial and from the open label Expanded Access Program, which together have demonstrated that vision loss can be mitigated or reversed in patients treated with Raxone®.
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