In a landmark move for the rare disease community, the U.S. Food and Drug Administration (FDA) has granted approval to Amgen’s monoclonal antibody therapy, Uplizna (inebilizumab) on April 3, 2025, for the treatment of Immunoglobulin G4-related disease (IgG4-RD) marking the first-ever FDA-approved treatment for this chronic and often misdiagnosed immune disorder.
Originally developed by Viela Bio and first approved by the FDA in June 2020 for Neuromyelitis Optica Spectrum Disorder (NMOSD), Uplizna was later acquired by Horizon Therapeutics, and subsequently became part of Amgen’s portfolio following Amgen’s acquisition of Horizon in 2023.
This latest approval marks the first-ever FDA-approved therapy for IgG4-RD, a chronic condition where the immune system becomes overactive, leading to inflammation and fibrotic damage in organs like the pancreas, liver and kidneys. Affecting approximately 20,000 people in the U.S., IgG4-RD has traditionally been managed with corticosteroids, which often come with significant side effects and long-term risks.
Uplizna, administered as an intravenous infusion every six months following two initial loading doses, works by targeting and depleting CD19-positive B cells, a key driver in the disease’s inflammatory process. In clinical trials, the drug reduced disease flare-ups by an impressive 87% compared to placebo, offering new hope for patients in need of a safer, long-term treatment strategy.
The approval not only provides a much-needed alternative to steroids but also represents a major advancement in autoimmune disease therapy. With its previous approval for NMOSD, Uplizna is emerging as a promising treatment for severe immune-mediated conditions. This decision reinforces the growing emphasis on targeted biologic therapies and personalized approaches in managing rare diseases.
Experts believe the impact of Uplizna could extend well beyond its current indications, with projected global sales reflecting strong confidence in its clinical value.
Disclaimer: The content provided in this article is for informational purposes only and is not intended as a substitute for professional medical advice, diagnosis, or treatment. It is not a promotion or advertisement for any pharmaceutical product. The purpose of this article is solely to share recent updates on newly approved therapies. Always seek the guidance of a qualified healthcare provider with any questions you may have regarding a medical condition or treatment option.
