FDA grants orphan drug designation to neuromyelitis optica therapy
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation, which is reserved for therapies that can be used in the treatment of rare diseases, to investigational anti-CD19 monoclonal antibody MEDI-551. The treatment is a product of MedImmune, the global biologics research and development division of pharmaceutical corporation AstraZeneca.
“The orphan designation for MEDI-551 underscores the significant need for an effective medicine for NMO, a rare, devastating disease which causes increasing damage and disability with each attack,” Bing Yao, MedImmune's senior vice president of research and development, said. “MEDI-551 has a unique, targeted mechanism of action offering potential for the treatment of NMO. We look forward to working with the FDA to advance MEDI-551 to patients suffering from NMO as quickly as possible.”
There is no cure nor approved medications for NMO, which attacks the central nervous system and can be fatal. NMO patients' healthy cells are attacked by their own immune systems. The condition typically affects the optic nerves and spinal cord and is diagnosed in approximately five of every 100,000 people.
MEDI-551 is now in a phase IIb clinical trial.
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