AstraZeneca’s potential medicine for Neuromyelitis Optica Spectrum Disorder receives orphan designation in Europe

AstraZeneca and its global biologics research and development arm, MedImmune, today 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.1 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. We look forward to continuing to develop this potential new medicine to help treat patients with this rare and devastating disease.”

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 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).

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

– ENDS –

[1] National Institute of Neurological Disorders and Stroke, National Institutes of Health



About inebilizumab

Inebilizumab is a humanised, monoclonal antibody that binds with high affinity to CD19, a protein expressed on a broad range of B cells, including certain B cells called plasmablasts. Research has shown that autoantibodies called AQP4-Ab (or NMO-IgG) directed against the protein aquaporin-4 - produced in these plasmablasts - play a key role in NMO disease pathogenesis. Inebilizumab binds directly to CD19 protein on the surface of the plasmablasts and depletes them. Inebilizumab is currently being evaluated in a global clinical trial in Neuromyelitis Optica (NMO)/Neuromyelitis Optica Spectrum Disorders. More information can be found on


About Neuromyelitis Optica Spectrum Disorders (NMOSD)

Neuromyelitis optica spectrum disorder (NMOSD) is a recently proposed unifying term for neuromyelitis optica (NMO) - also known as Devic's disease - and related syndromes. NMOSD is a severe relapsing neuroinflammatory autoimmune disease that is rare and can be fatal. In NMOSD, immune system cells and antibodies attack cells in the optic nerves and spinal cord. The damage to the optic nerves produces swelling and inflammation that may cause pain and loss of vision; the damage to the spinal cord may cause weakness, paralysis, loss of sensation, problems with bladder and bowel function, and respiratory failure, with each attack leading to further damage and disability. It primarily affects women and it may be more common in non-Caucasians. There is currently no cure or approved treatment for NMO.


About MedImmune
MedImmune is the global biologics research and development arm of AstraZeneca, a global, innovation-driven biopharmaceutical business that focuses on the discovery, development and commercialization of small molecule and biologic prescription medicines. MedImmune is pioneering innovative research and exploring novel pathways across Oncology, Respiratory, Cardiovascular & Metabolic Diseases, and Infection and Vaccines. The MedImmune headquarters is located in Gaithersburg, Md., one of AstraZeneca’s three global R&D centres, with additional sites in Cambridge, UK and Mountain View, CA. For more information, please visit


About AstraZeneca

AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three main therapy areas - Oncology, Cardiovascular & Metabolic Diseases and Respiratory. The Company also is selectively active in the areas of Autoimmunity, Neuroscience and Infection. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. For more information, please visit and follow us on Twitter @AstraZeneca.


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