Uplizna

Uplizna is used to treat three autoimmune conditions in adults:

• neuromyelitis optica spectrum disorder (NMOSD) in people who are anti-aquaporin-4 (AQP4) antibody positive
• immunoglobulin G4-related disease (IgG4-RD)
• generalized myasthenia gravis (gMG) in people who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

It is given as an intravenous (IV) infusion, usually once every 6 months after a starting course of two infusions.

Uplizna is a CD19-directed cytolytic antibody. It works by targeting CD19, a marker found on immune cells called B lymphocytes (including pre-B and mature B cells). By binding to these cells, Uplizna triggers their destruction, which lowers the number of B cells and helps calm an overactive immune system. Exactly how this helps in NMOSD, IgG4-RD, and gMG is not fully known.

Uplizna (inebilizumab-cdon) first gained FDA approval on June 11, 2020, for NMOSD. Its approved uses were later expanded to include IgG4-RD and generalized myasthenia gravis in 2025. There is no generic or biosimilar version.

B lymphocytes (B cells) are white blood cells that help drive the immune response, including the production of antibodies that can attack the body's own tissues in autoimmune disease. Uplizna is a humanized monoclonal antibody that attaches to CD19, a protein on the surface of most B cells. Once attached, it flags those cells so the immune system destroys them, a process called antibody-dependent cellular cytolysis. This depletes B cells from the blood, which reduces the harmful immune activity behind NMOSD, IgG4-RD, and gMG. B-cell counts drop within days of an infusion and typically stay low for months.

Uplizna was studied in three placebo-controlled clinical trials, one for each approved condition:

• NMOSD (N-MOmentum trial, NCT02200770): Among anti-AQP4 antibody-positive adults, Uplizna reduced the risk of an NMOSD relapse by about 77% compared with placebo. During the roughly 6.5-month controlled period, a relapse occurred in 11% of people given Uplizna versus 42% given placebo, and hospitalizations were lower.
• IgG4-RD (MITIGATE trial, NCT04540497): Over 52 weeks, Uplizna reduced the risk of a disease flare by 87% compared with placebo (a flare occurred in about 10% versus 60%). More people on Uplizna reached flare-free, treatment-free remission at 1 year (57% versus 22%).
• gMG (MINT trial, NCT04524273): At 26 weeks, adults on Uplizna had greater improvement in daily-living symptoms than placebo, with an average 4.2-point drop in the MG-ADL score versus 2.2 points for placebo. Muscle strength (QMG score) also improved more with Uplizna.

Uplizna is given as an intravenous (IV) infusion by a healthcare provider.

• During the starting period, you receive 2 infusions given 2 weeks apart.
• Your next dose is a single infusion 6 months after the first, and then one infusion every 6 months after that.
• Each infusion takes about 90 minutes, and you will be monitored for at least 1 hour afterward.

Before each infusion, your provider will give premedications to lower the risk of an infusion or allergic reaction. These usually include a corticosteroid, an antihistamine, and acetaminophen.

Do not receive Uplizna if you have:

• had a life-threatening infusion reaction to Uplizna
• an active hepatitis B virus infection
• active or untreated inactive (latent) tuberculosis

Before receiving Uplizna, tell your healthcare provider about all of your medical conditions, including if you:

• have or think you have an infection
• have ever taken, currently take, or plan to take medicines that affect your immune system, or other treatments for NMOSD, IgG4-RD, or gMG
• have or have ever had hepatitis B, or are a carrier of the hepatitis B virus
• have or have ever had tuberculosis
• have had a recent vaccination or are scheduled to receive any vaccinations
• are pregnant or plan to become pregnant
• are breastfeeding or plan to breastfeed

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

Pregnancy

Uplizna may harm an unborn baby based on animal studies. Females who can become pregnant should use effective birth control during treatment and for 6 months after the last infusion. If you become pregnant while receiving Uplizna, you are encouraged to enroll in the pregnancy exposure registry by calling 1-303-724-4644 or visiting www.upliznapregnancyregistry.com. See Uplizna pregnancy and breastfeeding warnings for more detail.

Breastfeeding

It is not known if Uplizna passes into breast milk. Talk to your healthcare provider about the best way to feed your baby if you receive Uplizna.

Most common side effects

The most common side effects depend on the condition being treated:

• NMOSD: urinary tract infections and joint pain (arthralgia)
• IgG4-RD: urinary tract infections and low white blood cell counts (lymphopenia)
• gMG: headache and infusion-related reactions

Serious side effects and warnings

Uplizna may cause the following serious side effects:

• Infusion reactions. Uplizna can cause infusion reactions, including anaphylaxis, that can be serious. You will be monitored during the infusion and for at least 1 hour after each dose. Tell your healthcare provider right away about symptoms such as headache, nausea, sleepiness, shortness of breath, fever, muscle aches, rash, or palpitations. Your provider may slow or stop the infusion and treat the symptoms.
• Infections. Serious infections can happen during treatment. Tell your healthcare provider right away if you have signs of infection such as painful or frequent urination, nasal congestion, runny nose, sore throat, fever, chills, cough, or body aches. Taking Uplizna with other medicines that weaken the immune system may raise this risk.
• Hepatitis B virus (HBV) reactivation. Your provider will do blood tests for hepatitis B before you start. If you have ever had hepatitis B, the virus can become active again during or after treatment, which may cause serious liver problems, including liver failure or death.
• Progressive multifocal leukoencephalopathy (PML). PML is a rare and serious brain infection that can lead to death or severe disability. Call your provider right away about symptoms such as weakness on one side of the body, loss of coordination, vision changes, or changes in thinking, memory, or personality.
• Tuberculosis (TB). Your provider will check whether you are at risk for TB or have had it before starting treatment.
• Low blood cell counts and low immunoglobulins. Uplizna can lower certain blood cells and antibody (immunoglobulin) levels over time. Your provider will do blood tests to monitor these.
• Vaccinations. Live or live-attenuated vaccines are not recommended during treatment. Complete any needed vaccines at least 4 weeks before starting Uplizna, and talk to your provider before any vaccination.

It is not known if Uplizna is safe or effective in children.

These are not all the possible side effects of Uplizna. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088. See Uplizna side effects for more detail.

The recommended dose of Uplizna for NMOSD, IgG4-RD, and gMG is the same:

• Initial dose: 300 mg IV infusion
• Two weeks later: a second 300 mg IV infusion
• Starting 6 months after the first infusion: a single 300 mg IV infusion every 6 months

Each dose is diluted in 250 mL of 0.9% sodium chloride before it is given. See detailed Uplizna dosage information.

Tell your doctor about all your other medicines, especially medicines that weaken the immune system, such as cancer medicines, corticosteroids and other steroids, and medicines used to prevent organ transplant rejection. Using these with Uplizna may increase your risk of infection.

Because Uplizna is not broken down by the liver's CYP450 enzymes, it is unlikely to have the kind of interactions seen with many oral medicines. Live or live-attenuated vaccines should not be given during treatment. This list is not complete, and other medicines may interact with Uplizna. See Uplizna drug interactions for more detail.

Active ingredient: inebilizumab-cdon

Inactive ingredients: L-histidine, L-histidine hydrochloride monohydrate, polysorbate 80, sodium chloride, alpha,alpha-trehalose dihydrate, and water for injection.

Uplizna is supplied as a 100 mg/10 mL (10 mg/mL) solution in single-dose vials, in a carton of three vials. It should be stored in a refrigerator at 36 degrees F to 46 degrees F (2 degrees C to 8 degrees C) in the original carton to protect it from light. Do not freeze or shake, and store the vials upright.