Viela Bio Announces U.S. FDA Approval of UPLIZNA? (inebilizumab-cdon) for the Treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD)
UPLIZNA? is the first and only B cell depleter approved for the treatment of NMOSD in adults who are anti-aquaporin-4 (AQP4) antibody positive
GAITHERSBURG, Md., June 11, 2020 (GLOBE NEWSWIRE) -- Viela Bio (Nasdaq:VIE) today announced that the U.S. Food and Drug Administration (FDA) has approved?UPLIZNATM?(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%1?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,?UPLIZNATM?a humanized CD19-directed monoclonal antibody?significantly reduced the risk of attacks and also reduced hospitalizations when given as a monotherapy,? said Bruce Cree, M.D., Ph.D., 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. ?UPLIZNATM?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 NMOSD2. Multiple lines of evidence suggest that NMOSD is a B-cell-mediated disorder. ?As an organization that understands and represents the struggle of patients and their loved ones affected by NMOSD, we are pleased that now there is another treatment option that could reduce their attacks, which can lead to devastating and irreversible disability,? said Victoria Jackson, co-founder of the Guthy-Jackson Charitable Foundation, a non-profit organization dedicated to funding research and raising awareness about NMOSD. ?We have been proud to partner with Viela Bio and congratulate them and the NMOSD community on this important milestone.? The approval of?UPLIZNATM?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.?UPLIZNATM?also demonstrated statistically significant benefits in key secondary endpoints, including reductions in NMOSD-related hospitalizations. Additionally,?UPLIZNATM?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?). Commented Bing Yao, Ph.D., Chief Executive Officer at Viela Bio: ?We are proud that Viela Bio?s first approved medicine has the potential to help thousands of patients with NMOSD, a progressive and debilitating neuroinflammatory disease. We are incredibly grateful to the patients, families and care partners who participated in and supported our research.? Viela anticipates product launch in June. Study Design Overview and Efficacy Results Summary Patients in the N-MOmentum trial were randomized 3:1 (UPLIZNATM?to placebo) to receive two introductory doses of 300 mg of?UPLIZNATM?monotherapy or placebo at Day 1 and Day 15. The patients were followed for a total of 197 days. Following that randomized-controlled period (RCP), patients were given the option to enter an open-label extension period, in which every participant received 300 mg of?UPLIZNATM?monotherapy every 6 months. The study was concluded early on the recommendation of the independent data monitoring committee, based on evidence of efficacy. Results from the anti-AQP4 antibody positive patient subgroup are shown in the chart below.Treatment Group | ||
UPLIZNATM N = 161 | Placebo N = 52 | |
Time to Adjudication Committee-Determined Relapse (Primary Efficacy Endpoint) | ? | |
Number (%) of patients with relapse | 18 (11.2%) | 22 (42.3%) |
Hazard ratio (95% CI)a | 0.227 (0.121, 0.423) | |
p-valuea | <?0.0001 |
- A history of life-threatening infusion reaction to?UPLIZNATM
- Active hepatitis B infection
- Active or untreated latent tuberculosis