AstraZeneca Drug Receives Orphan Status for Rare Inflammatory Disease

AstraZeneca’s biologics research and development subsidiary, MedImmune has been granted Orphan Drug Designation by the FDA for its investigational anti-CD19 monoclonal antibody, MEDI-551, for treatment of patients with neuromyelitis optica (NMO) and also neuromyelitis optica spectrum disorders (NMOSD).

MEDI-551 is currently in Phase IIb clinical development for NMO (which is also known as Devic’s syndrome): a rare and potentially fatal autoimmune disease of the central nervous system, where the immune system attacks healthy cells. The disease most commonly affects the optic nerves and spinal cord, resulting in severe damage. NMO causes severe muscle weakness and paralysis, loss of vision, respiratory failure, problems with bowel and bladder function and neuropathic pain.

Research has shown that patients with NMO and NMOSD develop antibodies against a protein in their body called aquaporin-4, and these antibodies play a key role in NMO disease pathogenesis. MEDI-551 directly targets and depletes cells that produce these antibodies.

MEDI-551 is currently being evaluated in a global clinical trial in Neuromyelitis Optica (NMO) and Neuromyelitis Optica Spectrum Disorders, and is also being studied in a Phase II trial in diffuse B-cell lymphoma.

If approved, MEDI-551 would be the first drug to have been so for NMO, which affects approximately five in 100,000 people. The FDA grants orphan drug status to promising new medicines for conditions that affect less than 200,000 people in the US.

 “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. 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.” States Bing Yao, senior vice president, R&D and head of the Respiratory, Inflammation and Autoimmunity Innovative Medicines unit at MedImmune.


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