Uplizna (Inebilizumab-cdon)

Uplizna (inebilizumab-cdon) was developed by Viela Bio (now part of Horizon Therapeutics) as a treatment for people with neuromyelitis optica spectrum disorder (NMOSD). The therapy is designed to lower the risk of relapse among patients following an NMOSD autoimmune attack, which typically affects the optic nerves and spinal cord.

The U.S. Food and Drug Administration (FDA) approved Uplizna in June 2020 for adults with NMOSD who test positive for aquaporin-4 water channel autoantibodies (AQP4-IgG). Patients receive Uplizna as an intravenous (IV; into-the-vein) infusion twice a year.

Viela Bio entered into a collaboration with Mitsubishi Tanabe Pharma in October 2019 to commercialize Uplizna in Japan and eight other Asian regions.

How does Uplizna work?

NMOSD is an autoimmune disorder in which the body mistakenly attacks its own cells. The majority of patients with NMOSD have high levels of antibodies that target glial cells — cells that support the nervous system.

Specialized cells of the immune system called B cells produce antibodies. When the antibodies bind to glial cells, they trigger an immune chain reaction called the complement pathway, which leads to inflammation and damage to the glial cells and surrounding nerve cells, causing the symptoms of NMOSD.

Uplizna is a humanized anti-CD19 monoclonal antibody that targets B cells. When Uplizna binds to the B cells, it inhibits their antibody production, reducing the risk of NMOSD attacks.

Uplizna in clinical trials

Researchers tested the safety and effectiveness of Uplizna in a double-blind, randomized, placebo-controlled, Phase 2/3 trial, called N-MOmentum (NCT02200770). The trial recruited 230 patients with NMOSD, who were randomly selected, in a 3:1 ratio, to receive Uplizna or a placebo. Neither the researchers nor the participants knew which patients were receiving the medication and which the placebo.

The participants received 300 mg of Uplizna or a placebo through an IV infusion on days 1 and 15 of the study, after which they were monitored for 197 days (about 6.5 months). After this period, all patients had the option to enter the open-label portion of the experiment to receive 300 mg of Uplizna every six months. The primary endpoint of the trial was the time it took until the participants had the first relapse.

The results of the trial, published in the medical journal The Lancet, showed that patients receiving Uplinza had a much lower risk of relapse compared with those receiving the placebo. A total of 21 of the 174 patients (12%) receiving Uplizna had an attack compared with 22 of the 56 (39%) receiving the placebo. Of the 230 patients enrolled, 213 were positive for AQP4-IgG autoantibodies while the other 17 tested negative. Among those who were AQP4-IgG positive, 89% receiving Uplizna were relapse-free after six months, compared with 58% of those receiving the placebo.

Additional analyses of the study, including data from the ongoing open-label portion, appeared in the journal Drugs. That data showed that, after a year of the open-label portion of the study, 85% of the 216 patients who continued receiving Uplizna were free of attacks. Scores using the expanded disability status scale (EDSS) also improved for patients after at least one year of treatment.

The study is ongoing, and is expected to be completed in October 2022.

Other information

Researchers also are investigating Uplizna as a treatment for kidney transplant desensitization, myasthenia gravis, and IgG-4-related disease.

Uplizna may cause serious side effects, including infusion reactions, infections, and low white blood cell counts. The most common side effects include urinary tract infections and joint pain.


Last updated: Dec. 3, 2020


Neuromyelitis News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.