FDA grants Orphan Drug Designation to AstraZeneca’s NMO medicine

AstraZeneca and MedImmune announced last week that the U.S. Food and Drug Administration (FDA) has granted MEDI-551, an anti-CD19 monoclonal antibody, Orphan Drug Designation.

MedImmune, a worldwide biologics research and development subsidiary of AstraZeneca, developed MEDI-551 to treat patients with neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorders (NMOSD). Medi-551 is now in Phase IIb clinical development for NMO.

NMO is a serious autoimmune disease of the central nervous system that causes the body’s immune system to attack healthy cells. The disease is most common in the optic nerves and spinal cord and can inflict serious damage. The symptoms of NMO include severe muscle weakness and paralysis, vision loss, respiratory system failure, functional problems in the bowels and bladder and neuropathic pain. NMO affects nearly five out of every 100,000 people and there is no current cure or approved medications for the disease.

Research of NMO and NMOSD has shown that the diseases cultivate antibodies to counter aquaporin-4, a protein produced by the body. These antibodies are a key component in the disease pathogenesis found in NMO. MEDI-551 is designed to seek out and destroy the cells that create these antibodies.

The Orphan Drug Designation grants orphan status to potential medicines to treat rare diseases or disorders that affect fewer than 200,000 people in the U.S.