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Temporary authorisation is provided by French national regulatory agency (ANSM) for novel LHON treatment

A Swiss based company, Santhera Pharmaceuticals Holding AG (SIX: SANN), has reported that the French National Agency for Medicines and Health Products Safety (ANSM) has granted an “autorisation temporaire d’utilisation dite de cohorte” (cohort ATU), for the company’s “Raxone” drug, a proposed novel treatment for Leber’s hereditary optic neuropathy (LHON). In a press release the company stated that the temporary authorization system allows patients in France to receive reimbursed treatment with a product before a marketing authorization is granted in the European Union. Idebenone, the active pharmaceutical ingredient of Raxone, is a synthetic short-chain benzoquinone compound and a cofactor for the enzyme NAD(P)H. According to the company, the 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”. The company is understood to be preparing a second marketing authorization approval document for submission to regulators sometime in Q12014.