Orphan status for Alexion's Solaris

US drugmaker Alexion Pharmaceuticals (Nasdaq: ALXN) says that Soliris (eculizumab), the company’s first-in-class terminal complement inhibitor, has received a positive opinion for orphan medicinal product designation from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) for the treatment of neuromyelitis optica (NMO), a life-threatening, ultra-rare neurological disorder.

The positive opinion of the COMP has now been forwarded to the European Commission for final approval and publication in the community register. Soliris is not approved in any country for the treatment of patients with NMO.

“We are pleased that the COMP has provided a positive opinion regarding orphan medicinal product status for Soliris for the treatment of patients with NMO, particularly after having been recently granted orphan-drug designation by the FDA,” said Martin Mackay, executive vice president and global head of R&D at Alexion, noting that “NMO is an extremely rare, chronic and debilitating disease that can lead to paralysis, blindness and death, and there are no approved therapies for it. Therefore, this news represents another positive step toward meeting the needs of this underserved patient population in the EU.”