Raxone (idebenone) was recently approved by the Ministry of Health in Israel as a treatment for visual impairment in adolescent and adult patients with Leber’s hereditary optic neuropathy (LHON) – a mitochondrial disease that can cause blindness.
The announcement was made by the maker of Raxone, Santhera Pharmaceuticals; it is the first approval for the medicine in LHON outside Europe. Santhera received a European Commission’s marketing authorization for Raxone as a treatment for LHON in September 2015.
LHON is a hereditary genetic condition that presents predominantly in young adult males as rapid, painless loss of central vision, usually leading to permanent blindness within a few months of symptom onset.
LHON is caused by genetic mutations in the DNA located in mitochondria, which are the structures that provide the energy our cells need to function properly. The genetic defect in mitochondrial DNA may result in a decreased production of energy and in an increased production of damaging molecules known as “reactive oxygen species” (ROS). The process culminates in a progressive loss of visual acuity and blindness.
Raxone is an oral drug (given at 900 mg, as two tablets taken three times per day, alongside meals) developed to circumvent the mitochondrial defect, reduce ROS, restore cellular energy levels in retinal cells and promote recovery of visual acuity.
At the time of European Commission approval, Thomas Klopstock, MD, professor of neurology at the University of Munich and coordinator of the German network for mitochondrial disorders, mitoNET, said “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.”
“We are pleased that Raxone has received marketing authorization for the treatment of LHON in Israel, the first approval for the product outside of Europe,” Giovanni Stropoli, chief commercial officer Europe & ROW at Santhera, said in a press release. “As part of our strategy to provide treatment to patients in Europe and beyond, we have signed a collaboration agreement with Megapharm Ltd., a leading Israeli private pharma marketing company, who is committed to making Raxone available to patients with LHON in Israel.”