Alexion Pharmaceuticals Inc Receives Orphan Drug Designation for Treatment of Neuromyelitis Optica

Thursday, 27 Jun 2013 04:50pm EDT 

Alexion Pharmaceuticals Inc announced that Soliris (eculizumab), the Company`s 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 reduction in the frequency of relapses (recurring attacks) in patients with severe, relapsing NMO.1 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. 

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27 Feb 2015