AstraZeneca’s potential drug for neuromyelitis optica spectrum disorder receives EU orphan designati

AstraZeneca and its global biologics research and development arm, MedImmune, announced that the European Medicines Agency (EMA) has granted orphan designation to inebilizumab (formerly MEDI-551) for the treatment of neuromyelitis optica spectrum disorder (NMOSD).

Developed by MedImmune, inebilizumab is currently in Phase IIb clinical development for NMOSD.

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. NMOSD may cause severe muscle weakness and paralysis, loss of vision, respiratory failure, problems with bowel and bladder function and neuropathic pain.

There is currently no cure or approved medicine for NMOSD, which affects about five in 100,000 people.

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.

Orphan designation

Orphan designation is a status assigned to a medicine intended for use in rare diseases. To be granted orphan designation by the EMA, a medicine must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening and has a prevalence of less than five people in 10,000 in the European Union.

Additionally, the intended medicine must aim to provide significant benefit to those affected by the condition. Orphan designation provides companies with development and market exclusivity incentives for designated compounds and medicines. Orphan designation is conferred following a positive opinion by the EMA’s Committee for Orphan Medicinal Products (COMP).

US approval

In March 2016, AstraZeneca announced that inebilizumab had received orphan drug designation from the US Food and Drug Administration (FDA).

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. We look forward to continuing to develop this potential new medicine to help treat patients with this rare and devastating disease."