Alexion announced that it has received Orphan Drug Designation from Japan's Ministry of Health, Labor, and Welfare (MHLW) for Soliris (eculizumab) as treatment for patients with neurological disorder neuromyelitis optica (NMO).
Soliris is a first-in-class terminal complement inhibitor designed to reduce the destruction of red blood cells or hemolysis. The drug is currently approved for two ultra-rare disorders paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Both the European Commission and the U.S. Food and Drug Administration (FDA) awarded orphan status to Soliris earlier this year as treatment for the rare neurological disease Myasthenia Gravis (MG).
Neuromyelitis optica is caused by the binding of NMO-IgG antibody to astrocytes, which leads to uncontrolled complement activation and destruction of myelin-producing cells. This in turns inflicts severe damage to the central nervous system especially to the spinal cord and optic nerve. NMO leads to severe weakness, respiratory failure, paralysis, blindness, loss of bowel and bladder function, and premature death. Half of patients who experience relapsing NMO have been reported to sustain permanent severe disability, including blindness and paralysis within five years of disease onset. NMO presently has no approved treatments.
Dr. Martin Mackay, EVP and global head of R&D at Alexion, said, “The orphan drug designation for eculizumab for NMO highlights the significant need for an effective and innovative treatment option for patients in Japan suffering from this debilitating and life-threatening disease. By specifically inhibiting the terminal complement pathway, eculizumab has the potential to improve outcomes for patients with NMO.”
Enrollment is currently ongoing for patients with relapsing NMO in the placebo controlled, multinational trial of Soliris labeled as PREVENT (Prevention of Relapses and EValuation of Eculizumab in NMO Treatment).
The MHLW grants orphan status to drugs and devices that address serious diseases with high unmet medical need affecting less than 50,000 patients in Japan. The designation awards a range of benefits to the sponsor company, including priority review. Once Soliris is approved for its orphan indication by the MHLW, Alexion will gain 10 years of marketing exclusivity.