European Medicines Agency (EMA) has granted orphan designation to AstraZeneca’s and MedImmune’s inebilizumab (formerly MEDI-551) for the treatment of neuromyelitis optica spectrum disorder (NMOSD).
The drug is in Phase IIb clinical development for NMOSD by MedImmune.
NMOSD is a rare, life-threatening autoimmune disease of the central nervous system in which the body’s immune system attacks healthy cells, most commonly in the optic nerves and spinal cord, resulting in severe damage, the company explained.
There is currently no cure or approved medicine for NMOSD, which affects about five in 100,000 people.
Bing Yao, Senior Vice President, R&D and Head of the Respiratory, Inflammation and Autoimmunity Innovative Medicines unit, MedImmune, said: “The EMA’s orphan designation for inebilizumab underscores the significant unmet need for patients with NMOSD, who currently have no options and face increasing damage and disability with each attack.”
Research has shown that patients with NMOSD develop antibodies against a protein called aquaporin-4 in their optic nerve and spinal cord. These antibodies play a key role in NMOSD disease pathogenesis. Inebilizumab directly targets and depletes cells that produce these antibodies, the company said
In March 2016, inebilizumab had received Orphan Drug Designation from the US Food and Drug Administration (FDA).