Amgen’s Uplizna (inebilizumab-cdon) has been approved by the US Food and Drug Administration (FDA) as the first treatment for adults with immunoglobulin G4-related disease (IgG4-RD), a rare fibroinflammatory disease estimated to affect 20,000 people in the US.
IgG4-RD can impact almost any organ in the body and is characterised by periods of remission and unpredictable flares, which can cause permanent organ damage. The disease typically begins between the ages of 50 and 70 and is more likely to occur in men.
Uplizna is a humanised monoclonal antibody that reduces the number of cells thought to contribute to the underlying process of the disease. The drug is already approved in the US to treat the rare autoimmune disease neuromyelitis optica spectrum disorder (NMOSD).
The FDA’s latest decision was supported by results from the phase 3 MITIGATE trial, in which Uplizna demonstrated an 87% reduction in the risk of IgG4-RD flare compared to placebo during a 52-week placebo-controlled period.
The drug was also associated with a reduction in annualised flare rate, at 0.10 for Uplizna-treated patients versus 0.71 for those receiving placebo, and showed significant improvements in flare-free, treatment-free complete remission, as well as flare-free, corticosteroid-free complete remission.
Jay Bradner, executive vice president of research and development at Amgen, said: “The FDA approval of Uplizna marks a significant turning point for IgG4-RD patients and physicians, who now have a proven treatment that targets a key driver of the disease, reducing the risk of flares and reliance on harmful long-term steroid use.”
Beyond IgG4-RD and NMOSD, Uplizna has also been granted orphan drug designation for generalised myasthenia gravis (gMG), a rare autoimmune disorder that impairs neuromuscular communication and can cause muscle weakness, trouble breathing, difficulty swallowing and impaired speech and vision.
Regulatory filing activities for Uplizna in gMG are underway, with submissions expected to be complete in the first half of 2025, according to Amgen.
“We are proud to deliver a therapy that has the potential to significantly improve care for patients with IgG4-RD and remain encouraged by Uplizna’s broader potential in other immune-mediated diseases, including NMOSD and gMG,” Bradner said.
