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Targeted therapies for NMOSD in development

Dr Dean Wingerchuk, Mayo Clinic, Rochester, United States
Phase 3, PREVENT

Neuromyelitis optica spectrum disorder (NMOSD) is a rare, relapsing, autoimmune, inflammatory disorder that typically affects the optic nerves and spinal cord and therefore causes blindness and paralysis. NMOSD relapses can cause significant and irreversible neurologic disability. No approved therapies exist for NMOSD. However, some targeted therapies are in development and promising results recently presented or published.

Eculizumab in AQP4-IgG-positive NMOSD

At least two thirds of NMOSD cases are associated with aquaporin-4 antibodies (AQP4-IgG) and complement-mediated damage to the central nervous system. Eculizumab, a terminal complement inhibitor, reduced the risk of NMOSD relapse in patients with AQP4-IgG-positive NMOSD. In the phase 3 PREVENT trial, 143 adults were randomised to receive intravenous eculizumab or placebo. The mean annualised relapse rate (ARR) in the 2 years before enrolment was 1.99. It was permitted to cont...

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