Amgen’s UPLIZNA Gains FDA Nod for Rare Inflammatory Disease Indication
The monoclonal antibody therapy is now the first and only treatment approved for patients with IgG4-RD.
Biotechnology innovator, Amgen, announced on April 3, 2025 that the U.S. FDA has approved its humanized monoclonal antibody (mAb) therapy, inebilizumab-cdon (UPLIZNA), as a treatment for adults with immunoglobulin G4-related disease (IgG4-RD) (1). This regulatory approval marks the first for IgG4-RD patients, meeting an unmet clinical need.
IgG4-RD is a progressive, chronic, systemic, immune-mediated, fibroinflammatory disease, which can affect a number of organs over a period of time. The disease is characterized by periods of remission and flares, and it has the potential to cause permanent organ damage whether symptoms are present or not. It is believed that CD19-expressing (CD19+) B cells drive inflammatory and fibrotic processes and also interact with other immune cells to contribute to IgG4-RD disease activity.
Inebilizumab-cdon works by depleting the key cells that contribute to the underlying disease process — autoantibody-producing CD19+ B cells — in a targeted and sustained way. While the precise therapeutic mechanism of inebilizumab-cdon is unknown, the therapy has been demonstrated, through the MITIGATE clinical trial, to reduce the risk of flares by 87% when compared with placebo in adults with IgG4-RD.
"Targeting CD19+ B cells with UPLIZNA has proven to be a highly effective approach to help address the pathophysiology of IgG4-RD,” said John Stone, M.D., M.P.H., principal investigator, and a professor of medicine at Harvard Medical School and the Edward A. Fox Chair in Medicine at the Massachusetts General Hospital, in the press release (1). “The clinical community now has an FDA-approved therapeutic innovation for patients that targets underlying disease mechanisms and helps to control disease activity by reducing flares in IgG4-RD.”
“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,” added Jay Bradner, M.D., executive vice president of Research and Development at Amgen, in the press release (1). "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 neuromyelitis optica spectrum disorder and generalized myasthenia gravis.”
Inebilizumab-cdon has already been approved by the FDA in 2020 as a treatment for adult patients with anti-aquaporin-4 antibody positive neuromyelitis optica spectrum disorder (2). Furthermore, the FDA has granted Orphan Drug Designation to inelbilizumab-cdon for the treatment of generalized myasthenia gravis and the company has regulatory filings underway for this indication, which are expected to be complete in the first half of 2025 (3).
References
Amgen. UPLIZNA (Inebilizumab-cdon) is Now the First and Only FDA-Approved Treatment for IgG4-Related Disease. Press Release, April 3, 2025.
FDA. FDA Approves New Therapy for Rare Disease Affecting Optic Nerve, Spinal Cord. Press Release, June 11, 2020.
Amgen. UPLIZNA (Inebilizumab-cdon) Significantly Improves Generalized Myasthenia Gravis Symptoms in Acetylcholine Receptor Autoantibody-Positive Patients Over 52 Weeks. Press Release, March 13, 2025.