FDA

Uplizna now approved for immunoglobulin G4-related disease

April 10, 2025

FDA approved Uplizna (inebilizumab-cdon), a CD-19 directed cytolytic antibody, for the treatment for adults with immunoglobulin G4-related disease (IgG4-RD).

Uplizna had previously been approved for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti-aquaporin-4 (AQP4) antibody positive.

Efficacy

Approval of the expanded indication was based on data from the phase MITIGATE trial (NCT04540497) that evaluated inebilizumab in 135 adults with newly diagnosed or recurrent IgG4-RD who required glucocorticoid (GC) treatment and who had a confirmed history of organ involvement at any time during the course of disease.

Study participants were randomly assigned to receive IV inebilizumab (n=68) or placebo (n=67). All patients were initially given 20-mg doses of GC therapy at the time of randomization and then began a 5mg taper q2wks until the end of 8 weeks. Primary endpoint was the time to first treated and adjudication committee (AC)-determined IgG4-RD flare during the 52-week randomized control period (RCP).

The time to the first treated and AC determined IgG4-RD flare was significantly longer in the Uplizna group, compared with the placebo group. Uplizna reduced the risk of treated and AC-determined IgG4-RD flare by 87%, compared with placebo (hazard ratio, 0.13; 95% confidence interval, 0.06-0.28; p < 0.0001).

For all patients in the trial, mean total GC use for IgG4-RD control per patient other than the planned GC taper was lower in the Uplizna-treated group vs. the placebo group, with a mean of 118.25 mg prednisone equivalent vs. 1384.53 mg prednisone equivalent, respectively during the RCP. Forty-two (62.7%) placebo-treated patients and seven (10.3%) Uplizna-treated patients received GC for IgG4-RD control other than the planned GC taper. The mean total GC use per patient for the 42 placebo-treated patients was 2202.76 mg prednisone equivalent and for the seven Uplizna-treated patients was 1148.71 mg prednisone equivalent.

Safety

The most common adverse reactions in IgG4-RD (at least 10% of patients treated with Uplizna and greater than placebo) were UTI and lymphopenia.

Sources:

Uplizna (inebilizumab-cdon) [package insert]. U.S. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/761142s003lbl.pdf Revised April 2025. Accessed April 9, 2025.

Uplizna® (inebilizumab-cdon) is now the first and only FDA-approved treatment for IgG4-related disease. [News release]. 2025. https://www.amgen.com/newsroom/press-releases/2025/04/uplizna-inebilizumabcdon-is-now-the-first-and-only-fdaapproved-treatment-for-igg4related-disease