The drug has been approved for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 antibody positive.
NMOSD is a rare neuroinflammatory autoimmune disease characterized by unpredictable attacks that often lead to severe, irreparable disability including loss of vision, paralysis, loss of sensation, bladder and bowel dysfunction, nerve pain and respiratory failure.
Each NMOSD attack can lead to further damage and disability.
Uplizna is a humanized monoclonal antibody targeting CD19+ B cells. Inebilizumab-cdon specifically binds to CD19, a cell surface antigen present on pre-B and mature B cell lymphocytes.
B cells are believed to play a central role in the pathogenesis of NMOSD.
PantheRx Rare Pharmacy, the largest independent and fastest growing specialty pharmacy in the United States, transforms lives by delivering medicine breakthroughs, clinical excellence, and access solutions to patients afflicted with rare and devastating conditions.
Although the incidence is as common as diabetes, less than 7% of the 7,000 known rare and devastating disorders have an approved therapy.
Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community.
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