Viela Bio announced the FDA approval of Uplizna (inebilizumab-cdon) for the treatment of adult patients with neuromyelitis optica spectrum disorder (NMOSD). The monotherapy drug is the first and only B cell-depleting antibody approved for the disease.
Uplizna is indicated for those 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.
“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, Uplizna—a humanized CD19-directed monoclonal antibody—significantly reduced the risk of attacks and also reduced hospitalizations when given as a monotherapy,” 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, said in a company news release. “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.”
NMOSD is a rare, severe, neuroinflammatory autoimmune disease that attacks the optic nerve, spinal cord and brain stem. In addition to potentially irreversible blindness and paralysis, patients may also experience loss of sensation, bladder and bowel dysfunction, nerve pain and respiratory failure. It is estimated that there are approximately 10,000 people in the U.S. suffering from NMOSD. Multiple lines of evidence suggest that NMOSD is a B-cell-mediated disorder.
The 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 6-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 2/3 trial”).
Viela anticipates product launch in June.