Santhera Pharmaceuticals is launching Raxone for the treatment of Leber's Hereditary Optic Neuropathy (LHON) in Germany, its first and largest EU market.
LHON is a rare inherited mitochondrial disease that usually leads rapidly to profound and permanent blindness.
Santhera chief commercial officer for Europe Giovanni Stropoli said: "The introduction of Raxone in Germany is a major step forward for LHON patients who now have an effective treatment option.
"We are particularly proud as Raxone is also the first approved treatment for any mitochondrial disease, a therapeutic area which is in the focus of our Company. Germany is our first and largest EU market but product availability is anticipated in additional countries in the immediate future."
"Mitochondrial disease treatments have been elusive for a long time. The availability of Raxone for patients with LHON is changing the paradigm and also raises hopes for other mitochondrial disorders," said Thomas Klopstock, MD, Professor of Neurology at the University of Munich, LHON researcher and coordinator of the German network for mitochondrial disorders, mitoNET.
"LHON patients usually become blind in young adulthood, which entails not only a personal and medical, but also a socioeconomic burden. With Raxone, we can now offer a treatment option which greatly improves the chances for significant recovery of visual acuity."
"As an organization representing the interests of patients affected with LHON, we are very excited that an approved medication is finally available for the treatment of this devastating disease," emphasized Claus-Peter Eisenhardt, President of the German Patient Advocacy of patients with mitochondrial diseases, within the German Society of Muscular Diseases (DGM).
"We are grateful to the European regulatory authorities, who, after careful evaluation of all available data, approved Raxone and made LHON a treatable disease."
Raxone is an oral medication, 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.