FDA Approves Inebilizumab for the Treatment of Neuromyelitis Optica Spectrum Disorder

The US Food and Drug Administration (FDA) has approved inebilizumab-cdon (Uplizna; Viela Bio, Gaitherburg, MD) for the treatment of adults with neuromyelitis optica spectrum disorder (NMOSD). The treatment will be available for individuals who are antiAQP4 antibody positive for antibodies to aquaporin 4 (AQP4) as a twice-a-year maintenance regimen following initial doses. Almost 80% of all individuals with NMOSD test positive for antiAQP4 antibodies.

“NMOSD is an extremely challenging disease to treat. Patients experience unpredictable attacks that can lead to permanent disability from blindness and paralysis. In addition, each subsequent attack may result in a cumulative worsening of disability. In the pivotal N-MOmentum trial (NCT02200770), Uplizna, a humanized CD19-directed monoclonal antibody, significantly reduced the risk of attacks and also reduced hospitalizations when given as a monotherapy,” said Bruce Cree, MD, PhD, MAS, the lead investigator for the N-MOmentum trial and professor of Clinical Neurology at the University of California San Francisco Weill Institute for Neurosciences. “Uplizna is an important new treatment option that provides prescribing physicians and patients living with NMOSD a therapy with proven efficacy, a favorable safety profile and a twice-a-year maintenance dosing schedule.”

The FDA approval of inebilizumab-cdon, previously received breakthrough therapy and orphan drug designations from the FDA, is based partially on the pivotal N-MOmentum trial results. The largest study ever conducted in a real-world spectrum of adults with NMOSD, which enrolled 213 antiAQP4 antibody positive participants and 17 antiAQP4 antibody negative participants. The global, placebo-controlled study demonstrated a significant reduction in risk of NMOSD attacks. Specifically, 89% of participants in the antiAQP4 antibody positive group remained relapse-free during the 6-month period post-treatment, compared with 58% of the participants taking placebo. 

Uplizna demonstrated a favorable safety and tolerability profile. Across both the randomized and open-label treatment, the most common adverse reactions (greater than 10%) were urinary tract infection (20%), nasopharyngitis (13%), infusion reaction (12%), arthralgia (11%), and headache (10%).