Cheshire’s Alexion Pharmaceuticals said Japanese regulators have designated its drug Soliris as an orphan drug for treatment of an ultra-rare disorder that damages the spine and optic nerve.
Orphan drug status in Japan can come with subsidies, priority review from regulators, tax credits and other advantages.
Neuromyelitis optica, or NMO, is characterized by weakness, paralysis, respiratory failure, blindness, loss of bowel and bladder function, and premature death, according to Alexion, which said half of patients suffer severe, permanent disability within five years of disagnosis.
Alexion is enrolling patients in a registration study that will evaluate the clinical benefits of Soliris in NMO patients.
Soliris already has orphan status for NMO in the United States and Europe.
