UPLIZNA Receives FDA Authorization for Myasthenia Gravis Treatment in Adults

Amgen disclosed that U.S. regulatory authorities have green-lit UPLIZNA (inebilizumab-cdon) as a therapeutic solution for adults suffering from generalized myasthenia gravis who test positive for anti-acetylcholine receptor or anti-muscle specific tyrosine kinase antibodies. This targeted immunotherapy represents a significant advancement for patients, delivering the prospect of sustained disease management through a simplified dosing regimen—just two annual maintenance injections following an initial loading phase.

Understanding the Disease and Treatment Mechanism

Generalized myasthenia gravis constitutes a rare, unpredictable B-cell-mediated autoimmune condition characterized by disrupted neuromuscular transmission, leading to fluctuating muscle weakness and fatigue. The disease pathophysiology centers on autoantibodies produced by CD19+ B cells, particularly plasmablasts and plasma cells, which attack essential neuromuscular junction proteins. The patient population in the United States spans approximately 80,000 to 100,000 individuals, many of whom have limited therapeutic options.

UPLIZNA works by targeting CD19+ B cells, effectively disrupting the production of pathogenic autoantibodies that drive myasthenia gravis progression. The mechanism directly addresses the underlying immune dysfunction rather than merely managing symptoms.

Expanding Approval History

This authorization marks the third indication for UPLIZNA within the FDA’s regulatory framework. The drug previously received approval in June 2020 for treating anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder in adults, followed by authorization in April 2025 for immunoglobulin G4-related disease. This growing approval portfolio demonstrates the drug’s versatility across multiple B-cell-mediated autoimmune disorders.