EU approval for Santhera's Raxone in LHON

The European Commission has granted marketing authorization for Raxone (idebenone) from Swiss specialty pharma company Santhera Pharmaceuticals (SIX: SANN).

Raxone becomes the first approved medicine available in all 28 member states of the European Union, Norway, Iceland and Liechtenstein 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 which, if untreated, usually leads to rapid, profound and permanent blindness in otherwise healthy patients.

"Our data demonstrate that Raxone treatment can prevent patients from further vision loss and can promote clinically relevant recovery of vision," said Thomas Meier, chief executive of Santhera, adding: "The European approval of Raxone as an efficacious treatment for LHON and as the first approved medication for a mitochondrial disease is a major milestone for Santhera as it marks our transition to a pharmaceutical company with a product on the market.”

“Major breakthrough in mitochondrial disease treatment”

"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," said Thomas Klopstock, Professor for Neurology at the University of Munich, LHON researcher and coordinator of the German network for mitochondrial disorders, mitoNET. "LHON is a particularly devastating condition because sufferers, who are otherwise healthy and often young, rapidly become bilaterally blind within a few months. Most will remain permanently blind if untreated," he noted.