On June 11, 2020, the Food and Drug Administration (FDA) approved a biologics license application (BLA) for Uplizna (inebilizumab-cdon; Viela Bio) for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.
NMOSD is an autoimmune disorder characterized by inflammation of the optic nerve and spinal cord. AQP4, a normal protein in the body, plays a role in NMOSD; approximately 70% of people with this disorder produce an autoantibody that binds to AQP4, leading to inflammation of the optic nerve and spinal cord. Uplizna is a CD19-directed cytolytic antibody that is administered via intravenous infusion.
The Uplizna BLA was supported by data from a randomized, placebo-controlled trial in 230 adult patients; 213 of the 230 patients had antibodies against AQP4 (anti-AQP4 antibody positive). During the 197-day study, the risk of an NMOSD relapse in the 161 anti-AQP4 antibody positive patients who were treated with Uplizna was reduced by 77% when compared with the placebo treatment group. There was no evidence of a benefit in patients who were anti-AQP4 antibody negative.
Food and Drug Administration (FDA). FDA Approves New Therapy for Rare Disease Affecting Optic Nerve, Spinal Cord [news release]. June 11, 2020. Available at: click here. Accessed June 12, 2020.