WASHINGTON (dpa-AFX) - Alexion Pharmaceuticals, Inc. (ALXN) said Soliris (eculizumab) has been granted orphan drug designation by Japan's Ministry of Health, Labour and Welfare or MHLW for the treatment of patients with neuromyelitis optica or NMO, a life-threatening, ultra-rare neurologic disorder.
In patients with NMO, chronic, uncontrolled complement activation results in severe damage to the central nervous system, predominantly impacting the optic nerve and spinal cord.
This disease is characterized by severe weakness, paralysis, respiratory failure, loss of bowel and bladder function, blindness and premature death.
The MHLW, based on the opinion of the Pharmaceutical Affairs and Food Sanitation Council, grants orphan status to drugs and medical devices that treat serious diseases of high unmet medical need which affect fewer than 50,000 patients in Japan.
This designation provides drug developers with certain benefits and incentives, including priority review for marketing authorization and a period of 10 years of market exclusivity if regulatory approval is received for the designated indication.
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