European Commission Grants Raxone® The First Medicine For Leber’s Hereditary Optic Neuropathy

Patricia Inacio, PhD avatar

by Patricia Inacio, PhD |

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Santhera Pharmaceuticals, a biopharmaceutical company devoted to the investigation and commercialization of medicines for treating mitochondria and neuromuscular diseases, recently announced that the European Commission granted Raxone® a market authorization in the European Union as the first medicine for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy.

Leber’s Hereditary Optic Neuropathy (LHON) is a genetic disease that causes blindness, most commonly in young men (onset is around patient teens or twenties). The disease is characterized by a sudden loss of central vision in one eye and proceeds to attack, within a few months after symptoms arise, the other eye, leading to permanent bilateral blindness.

LHON is caused by mutations in the DNA located in cellular structures called mitochondria – known as the cells’ powerhouses, since they are the responsible organelles inside cells for generating the energy that cells need to function. The defects in mitochondria DNA result in a decreased production of cells’ energy (called ATP), and an increase in production of damaging molecules (known as reactive oxygen species, ROS) culminating in a progressive loss of visual acuity and blindness.

Raxone® is an oral medication developed by Santhera Pharmaceuticals that showed great potential in treating LHON patients in the company’s randomized, placebo-controlled RHODOS clinical trial and from the open label Expanded Access Program. Both demonstrated that Raxone® administration significantly alleviated vision loss, and even reversed it in some patients.

Thomas Meier, PhD, Chief Executive Officer of Santhera Pharmaceuticals commented on the recent approved market authorization, “Our data demonstrate that Raxone treatment can prevent patients from further vision loss and can promote clinically relevant recovery of vision. 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. I personally want to thank the team that achieved this tremendous success and the patients and doctors who supported our clinical development program over the past years. We will now ensure patients have a rapid access to Raxone, with immediate availability in some European countries.”

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Thomas Klopstock, MD, Professor for Neurology at the University of Munich, LHON researcher and coordinator of the German network for mitochondrial disorders, mitoNET added, “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. 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.”