Drug to Treat Rare Mitochondrial Eye Disease Expanding to Eastern Europe

Santhera Pharmaceuticals recently announced an agreement with Ewopharma to make Raxone available for the treatment of a rare mitochondrial disease, known as Leber’s hereditary optic neuropathy (LHON), throughout Eastern Europe following the European Commission’s market authorization for the company’s oral drug.

Under the partnership, Raxone (idebenone) will soon be a treatment option for patients in Romania, Slovakia, Slovenia, Czech Republic, Hungary, Poland, Bulgaria and Croatia, as well as the Baltic countries, Lithuania, Latvia and Estonia. The drug is already available as a LHON treatment throughout most of Western Europe.

“As an organization historically present in Eastern European and Baltic countries with a specific focus on niche indications and diseases with high unmet medical need, we are very excited to have an opportunity to team up with Santhera to offer for the first time a treatment to patients with LHON,” said Alain Staub, Ewopharma’s CEO and chairman of the board, in a press release. “We will make every effort to introduce Raxone as quickly as possible in these countries.”

LHON is a genetic disease that often leads to blindness, and most frequently affects men, beginning in their teens or early 20s. The condition is caused by DNA mutations in cellular structures called mitochondria, known as the cells’ powerhouses since they are the organelles responsible for the generation of the energy that cells need to function properly. Defects in mitochondrial DNA result in a decreased production of cellular energy (ATP) and an increased production of damaging molecules (known as reactive oxygen species, ROS), and retinal ganglion cell dysfunction. The disease is painless but rapid, with permanent blindness usually resulting within months after the onset of symptoms.

Raxone is a synthetic short-chain benzoquinone and a co-factor for the enzyme NAD(P)H: quinone oxidoreductase (NQO1) that aims to circumvent the complex I defect, reduce and scavenge ROS, restore retinal ganglion cellular energy, and promote visual acuity. According to the release, the drug is the first treatment option available for LHON.

Raxone’s efficacy was evaluated in Santhera’s randomized, placebo-controlled, RHODOS trial and its open-label extension, which demonstrated that vision loss could be reversed or mitigated through treatment.

“The agreement with Ewopharma represents an important achievement for Santhera and underscores our commitment to making Raxone available to patients across all EU member states,” said Santhera’s CCO for Europe, Giovanni Stropoli. “We are confident that Ewopharma’s expertise in the field of rare diseases, coupled with their understanding of the region, will greatly benefit patients with this devastating disease.”