Inebilizumab, sold under the brand name Uplizna, is a medication for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults.^[8][9][5] Inebilizumab is a humanized mAb that binds to and depletes CD19+ B cells including plasmablasts and plasma cells.^[5]

The most common adverse reactions include urinary tract infection, headache, joint pain (arthralgia), nausea and back pain.^[8][5]

Inebilizumab was approved for medical use in the United States in June 2020,^[8][10] in the European Union in April 2022,^[7] and in Canada in December 2023.^[1] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.^[11]

Inebilizumab is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults with a particular antibody (patients who are anti-aquaporin-4 or AQP4 antibody positive).^[8][5]

Neuromyelitis optica spectrum disorder is a rare autoimmune disorder in which immune system cells and autoantibodies attack and damage the optic nerves and spinal cord.^[8] Neuromyelitis optica spectrum disorder can be associated with antibodies that bind to a protein called aquaporin-4 (AQP4). Binding of the anti-AQP4 antibody appears to activate other components of the immune system, causing inflammation and damage to the central nervous system.^[8] Clinically, the disease is manifested with attacks/relapses that result in neurological impairment such as blindness, paraplegia, sensory loss, bladder dysfunction, and peripheral pain. The disability from each attack is cumulative, making neuromyelitis optica spectrum disorder a chronically debilitating and potentially life-threatening disease.^[12]

The label for inebilizumab includes a warning for infusion reactions, potential depletion of certain proteins (hypogammaglobulinemia), and potential increased risk of infection — including progressive multifocal leukoencephalopathy, and potential reactivation of hepatitis B and tuberculosis.^[8][5]

The most common adverse reactions in the neuromyelitis optica spectrum disorder clinical trial were urinary tract infection, headache, joint pain (arthralgia), nausea and back pain.^[8]

Women who are pregnant should not take inebilizumab because it may cause harm to a developing fetus or newborn baby.^[8] The FDA advises health care professionals to inform females of reproductive age to use effective contraception during treatment with inebilizumab and for six months after the last dose.^[8]

Vaccination with live-attenuated or live vaccines is not recommended during treatment and should be administered at least four weeks prior to initiation of inebilizumab.^[8]

Inebilizumab was created from the research led by Thomas Tedder at Cellective Therapeutics,^[13] and development was continued by Viela Bio and MedImmune.^[14]

Inebilizumab was approved for medical use in the United States in June 2020.^[8][10]

The effectiveness of inebilizumab for the treatment of NMOSD was demonstrated in a clinical study (NCT02200770) of 230 adult participants that evaluated the efficacy and safety of intravenous inebilizumab.^[8] In the trial, 213 of the 230 participants had antibodies against AQP4 (anti-AQP4 antibody positive).^[8][10] During the 197-day study, the risk of an NMOSD relapse in the 161 anti-AQP4 antibody positive participants who were treated with inebilizumab was reduced by 77% when compared to the placebo treatment group.^[8] There was no evidence of a benefit in participants who were anti-AQP4 antibody negative.^[8] The primary efficacy endpoint was the time to the onset of the first adjudicated relapse on or before study day 197 evaluated by a blinded, independent, adjudication committee, who determined whether the attack met protocol-defined criteria.^[10] The trial was conducted at 82 sites in 24 countries (including the United States) in North and South America, Europe, Africa, Asia and Australia.^[10]

The U.S. Food and Drug Administration (FDA) granted the application for inebilizumab orphan drug designation and granted approval of Uplizna to Viela Bio.^[8]

In November 2021, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Uplizna, intended for the treatment of adults with neuromyelitis optica spectrum disorders (NMOSD) who are anti-aquaporin 4 immunoglobulin G (AQP4-IgG) seropositive.^[15] The applicant for this medicinal product is Viela Bio.^[15] Inebilizumab was approved for medical use in the European Union in April 2022.^[7][16]

Inebilizumab is the international nonproprietary name (INN) and the United States Adopted Name (USAN).^[17][18]

• Cree BA, Bennett JL, Kim HJ, Weinshenker BG, Pittock SJ, Wingerchuk DM, et al. (October 2019). "Inebilizumab for the treatment of neuromyelitis optica spectrum disorder (N-MOmentum): a double-blind, randomised placebo-controlled phase 2/3 trial". Lancet. 394 (10206): 1352–1363. doi:10.1016/S0140-6736(19)31817-3. PMID 31495497. S2CID 201839513.

• Clinical trial number NCT02200770 for "A Double-masked, Placebo-controlled Study With Open Label Period to Evaluate MEDI-551 in Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorders" at ClinicalTrials.gov