Biotechnology company Amgen has received authorization from the European Commission for UPLIZNA as an add-on therapy for adults with generalized myasthenia gravis (gMG) who test positive for anti-acetylcholine receptor or anti-MuSK antibodies.
The approval introduces a targeted treatment option for patients with the rare autoimmune neuromuscular disorder, which causes fluctuating muscle weakness and fatigue and can significantly impair daily activities. The therapy is designed to provide sustained disease control through an initial loading regimen followed by twice-yearly maintenance dosing.
Generalized myasthenia gravis is a subtype of myasthenia gravis estimated to affect between roughly 56,000 and 123,000 people across Europe. Because symptoms can worsen unpredictably and patients often rely on long-term steroid therapy, physicians have sought treatments that address the immune cause of the disease while reducing chronic steroid exposure.
Amgen said the approval was supported by results from the Phase 3 MINT clinical trial, the largest biologic study in gMG to include both antibody-positive subgroups. The trial incorporated a structured steroid-tapering strategy, allowing patients to gradually reduce prednisone use while monitoring disease control. By week 26, more than 87% of patients receiving the drug and about 85% receiving placebo had reduced steroid doses to low levels, suggesting symptom stability while tapering.
Company medical affairs leader Cesar Sanz Rodriguez said the therapy offers a first-in-class approach that may help reduce reliance on chronic steroid treatment. Neurologist John Vissing noted the drug works by selectively targeting CD19-positive B cells, which are believed to play a central role in disease activity.
The gMG authorization expands the medicine’s growing list of indications. The therapy was approved in Europe in 2025 for immunoglobulin G4-related disease and previously authorized for neuromyelitis optica spectrum disorder in multiple countries, including approvals from regulators in the United States, Canada and Brazil.
Industry analysts say targeted immune therapies are increasingly reshaping treatment for rare autoimmune diseases, replacing broad immunosuppression with biologics aimed at specific immune pathways. With twice-yearly dosing and potential long-term efficacy, Uplizna could become a significant new option for European patients seeking more consistent symptom control and fewer steroid-related complications.