CHESHIRE, Conn., Jun 27, 2013 (BUSINESS WIRE) — Alexion Pharmaceuticals, Inc. ALXN today announced that Soliris(R) (eculizumab), the company’s first-in-class terminal complement inhibitor, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of neuromyelitis optica (NMO), a life-threatening, ultra-rare neurological disorder. In a Phase 2 study presented at the 2012 annual meeting of the American Neurological Association (ANA), Soliris treatment was associated with a significant reduction in the frequency of relapses (recurring attacks) in patients with severe, relapsing NMO. Soliris is not approved for the treatment of patients with NMO.
The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.
“There are no approved therapies for patients with NMO, an extremely rare and debilitating disease that can lead to paralysis, blindness and death,” said Martin Mackay, Ph.D., executive vice president and global head of R&D at Alexion. “This orphan drug designation is a positive step toward understanding and meeting the needs of this underserved patient population. In clinical trials to date, terminal complement inhibition with Soliris appeared to significantly reduce the attack rate in patients with severe, refractory relapsing NMO.”