Viela Bio announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for the Company’s anti-CD19 monoclonal antibody inebilizumab, an investigational monotherapy for neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a rare, life-threatening autoimmune disease affecting the central nervous system.
“The Breakthrough Therapy Designation for inebilizumab is based on results from the largest monotherapy study ever conducted in NMOSD,” said Jorn Drappa, M.D., Ph.D., Chief Medical Officer and Head of Research & Development at Viela Bio. “Inebilizumab is a humanized monoclonal antibody designed to bind with high affinity to CD19 and deplete a broad range of B cells, including autoantibody-secreting plasmablasts and CD19-expressing plasma cells. We continue our efforts to bring inebilizumab to patients suffering from this devastating disease for which there are currently no approved medicines.”
Breakthrough Therapy Designation is designed to expedite the development and regulatory review of medicines intended to treat a serious condition that have shown encouraging early clinical results which may demonstrate substantial improvement on a clinically significant endpoint over available medicines. The designation for inebilizumab is based on data from the pivotal N-MOmentum study evaluating inebilizumab as monotherapy.
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The FDA and the European Medicines Agency granted Orphan Drug Designation for inebilizumab for the treatment of patients with NMOSD in March 2016 and March 2017, respectively.
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 treatment for NMOSD.
Breakthrough Therapy Designation (BTD) is a U.S. FDA program designed to expedite the development and review of drugs intended for serious or life-threatening conditions. In order to receive a BTD, preliminary clinical evidence must demonstrate that the drug may provide substantial improvement over currently available therapy on at least one clinically significant endpoint. The benefits of BTD include more intensive guidance from FDA on an efficient drug development program, access to a scientific liaison to help accelerate review time and organizational commitment from FDA.
NMOSD is a recently proposed unifying term for neuromyelitis optica (NMO)—also known as Devic's disease—and related syndromes. NMOSD is a rare, severe, relapsing, neuroinflammatory autoimmune disease that can be fatal. In NMOSD, about 80% of patients have autoantibodies to a water channel protein called aquaporin-4 (AQP4). These AQP4-IgG autoantibodies are thought to be produced by plasmablasts and plasma cells and bind primarily to astrocytes in the central nervous system. Binding of AQP4-IgG antibodies to central nervous system cells is believed to trigger attacks, which can damage the optic nerves, spinal cord and brain. Loss of vision, paralysis, loss of sensation, bladder and bowel dysfunction, nerve pain, and respiratory failure can all be manifestations of the disease. Each NMOSD attack leads to further damage and disability. NMOSD occurs more commonly in women and it may be more common in non-Caucasians. There is currently no cure or approved treatment for NMOSD.
Inebilizumab is a humanized monoclonal antibody that binds with high affinity to CD19, a protein expressed on a broad range of B cells, including antibody-secreting plasmablasts and some plasma cells. After binding to CD19, these cells are rapidly depleted from the circulation.