FDA approval of Uplizna for the treatment of neuromyelitis optica spectrum disorder – Viela Bio

Viela Bio announced that the FDA has approved Uplizna (inebilizumab-cdon) for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-AQP4 antibody positive as a twice-a-year maintenance regimen following initial doses. Approximately 80% of all patients with NMOSD test positive for anti-AQP4 antibodies.

The FDA approval of Uplizna—which previously received Breakthrough Therapy and Orphan Drug designations from the FDA—is based in part on results from the pivotal N-MOmentum trial, the largest study ever conducted in a real-world spectrum of adults with NMOSD. The global, placebo-controlled study—which enrolled 213 anti-AQP4 antibody positive patients and 17 anti-AQP4 antibody negative patients—met its primary endpoint by demonstrating a statistically significant reduction in risk of NMOSD attacks. Specifically, 89% of patients in the anti-AQP4 antibody positive group remained relapse-free during the six-month period post-treatment, compared to 58% of the patients taking placebo. Uplizna also demonstrated statistically significant benefits in key secondary endpoints, including reductions in NMOSD-related hospitalizations. Additionally, Uplizna demonstrated a favorable safety and tolerability profile.

Across both the randomized and open-label treatment in Study 1, the most common adverse reactions (greater than 10%) were urinary tract infection (20%), nasopharyngitis (13%), infusion reaction (12%), arthralgia (11%), and headache (10%). The results from the N-MOmentum trial were published in the peer-reviewed journal, The Lancet, in September 2019 (“Inebilizumab for the treatment of neuromyelitis optica spectrum disorder (N-MOmentum): a double-blind, randomised placebo-controlled phase II/III trial”).