FDA approves new therapy for rare disease affecting optic nerve, spinal cord

FDA on Thursday approved inebilizumab-cdon (Uplizna—Viela Bio) injection for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 or AQP4 antibody positive.

FDA on Thursday approved inebilizumab-cdon (Uplizna—Viela Bio) injection for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 or AQP4 antibody positive. Inebilizumab-cdon is only the second approved treatment for NMOSD, a rare autoimmune disease of the central nervous system that primarily affects the optic nerves and spinal cord. The condition is thought to affect about 4,000-8,000 patients in the United States. A clinical study involving 230 patients found that the risk of an NMOSD relapse in the 161 anti-AQP4 antibody positive patients who were treated with inebilizumab-cdon dropped by 77% when compared with placebo recipients. The drug's prescribing information includes a warning for infusion reactions, potential depletion of certain proteins, potential increased risk of infection, and potential reactivation of hepatitis B and tuberculosis. The most common adverse reactions in the clinical trial were urinary tract infection, headache, joint pain, nausea, and back pain. FDA awarded inebilizumab-cdon Orphan Drug designation.