In the Phase III MITIGATE trial, Uplizna cut IgG4-related disease flares by 87% versus placebo.
The FDA approved Amgen’s anti-CD19 therapy Uplizna Thursday for use in patients with IgG4-related disease. Uplizna is the “first and only treatment” approved for this indication, according to the pharma’s statement.
Uplizna’s label expansion is backed by data from the Phase III MITIGATE trial. Amgen first presented data from the study in November 2024, touting an 87% drop in the risk of disease-related flares versus placebo. Over 52 weeks of follow-up, seven of 68 Uplizna-treated patients experienced flares, as opposed to 40 of 67 placebo counterparts. Amgen called this effect highly statistically significant.
In addition, 57.4% of patients in the Uplizna arm achieved complete remission at 52 weeks, versus 22.4% in the placebo group. Nearly 90% of Uplizna-treated participants no longer needed glucocorticoid treatment, versus 37.3% of placebo patients. After just 8 weeks of treatment, the Uplizna group saw a tenfold reduction in total glucocorticoid use as opposed to placebo.
Designed to be delivered intravenously, Uplizna is a humanized monoclonal antibody that binds to the CD19 protein, typically found on the surface of certain immune cells, in turn depleting both immature and mature B cells. This mechanism allows Uplizna to target an underlying pathological pathway of IgG4-related disease, a condition characterized by organ damage due to massive infiltration of B cells into tissues.
In Amgen’s news release, R&D Head Jay Bradner called Uplizna’s expansion into IgG4-related disease a “significant turning point” for patients and physicians, “who now have a proven treatment that targets a key driver of disease.”
Uplizna is also approved for the treatment of neuromyelitis optica spectrum disorder and is being studied for generalized myasthenia gravis. Last month, Amgen posted 52-week Phase III findings, showing that Uplizna improved activities of daily living in a specific subset of patients, as well as easing disease activity versus placebo.
Amgen is planning to file for Uplizna’s approval in this indication, with a submission planned for the first half of this year.
Earlier this year, Amgen cleared another regulatory hurdle when its KRAS blocker Lumakras won the FDA’s blessing for use in colorectal cancer. The label expansion, which came in January, was a pleasant surprise to the pharma, especially since its Phase III CodeBreaK 300 trial failed to demonstrate a significant improvement in survival for Lumakras versus an investigator’s choice of treatment.
The FDA’s approval of Lumakras in colorectal cancer was based on progression-free survival findings—the “major efficacy outcome” of CodeBreaK 300, as per the regulator—which improved significantly in patients who were given Lumakras plus Vectibix. Lumakras is also approved for non-small cell lung cancer, as per its label.
