CHMP Recommends Marketing Authorization for Santhera’s Raxone for Patients with LHON

Santhera Pharmaceuticals, a Swiss specialty pharmaceutical company, recently announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended granting marketing authorization for Raxone (idebenone) to treat the visual impairment found in adult and adolescent patients who suffer from LHON.

LHON is a genetic mitochondrial condition that leads to profound, rapid, and often permanent blindness in patients who are healthy. Raxone® is the first approved treatment option for the mitochondrial condition.

“This is a major breakthrough as it paves the way for the first medicinal product to become available for the treatment of a mitochondrial disease,” stated Thomas Klopstock, MD (Professor for Neurology at the University of Munich, LHON investigator and coordinator of the German network for mitochondrial disorders, mitoNET). “LHON is a severe form of vision loss caused by mitochondrial dysfunction. Affected patients, usually young and otherwise healthy, rapidly lose central vision and become bilaterally blind within a few months from the onset of symptoms. Although there is a chance for partial or even full spontaneous recovery, most patients remain permanently blind if untreated. The mode of action of idebenone provides a clear biochemical and medical rationale and the clinical data demonstrate that vision of affected patients can substantially improve upon treatment with Raxone. This recommendation is a landmark in mitochondrial disease research worldwide and will undoubtedly spur further research in this direction.”

“We are very excited about the CHMP’s positive opinion, which recognizes the urgent medical need for a treatment for this devastating disease,” stated Thomas Meier, PhD, CEO of Santhera. “We can now execute on our plans to ensure Raxone is made available to patients in the EU as soon as the European Commission marketing authorization is received.”

The European Medicines Agency’s Committee for Medicinal Products for Human Use based the Raxone recommendation on positive results from the RHODOS trial, a randomized, placebo controlled study, and also based on comparative results of a case record study. The CHMP determined that all the results were enough to provide LHON an orphan designation, with no available therapies. For post-authorization measures, the company is gathering long-term safety and efficacy results for patients with LHON.

The European Commission will now look at the CHMP’s opinion of Raxone in order to make a final decision on EU-wide marketing authorization. Raxone has orphan designation which provides 10 years of market exclusivity from the date of EC approval.