FDA broadens label for Amgen’s Uplizna as it enters rare disease competition

The FDA has expanded the label for Uplizna (inebilizumab-cdon) to include treatment of generalized myasthenia gravis (gMG) in adults who are anti–acetylcholine receptor (AChR) and anti–muscle-specific tyrosine kinase (MuSK) antibody positive.3 4 6

This new gMG indication adds to Uplizna’s existing approvals for neuromyelitis optica spectrum disorder (NMOSD) in adults who are anti–aquaporin-4 (AQP4) antibody positive and for IgG4-related disease, broadening its rare disease label.1 7

Uplizna is the first and only CD19-targeted B‑cell therapy approved for gMG, positioning it in direct competition within the rare autoimmune neuromuscular disease market.3 4

The drug is a humanized, afucosylated IgG1 kappa monoclonal antibody that targets CD19-positive B cells to induce rapid, deep, and durable B‑cell depletion, aiming at a biological root cause of disease.1

In gMG, Uplizna is given as two initial loading doses followed by twice-yearly maintenance infusions, offering the potential for long-term disease control with relatively infrequent dosing.1 3

Phase 3 clinical data (MINT trial) showed significant improvements in MG-ADL and QMG scores with a tolerable safety profile, supporting FDA approval in gMG.1 5

Amgen reported Uplizna sales of $379 million in full-year 2024 and $422 million in the first nine months of 2025, and the new gMG indication is expected to further grow revenue as it competes in the rare disease segment.2

Company leadership highlighted that the gMG approval is a “significant advancement” for patients, emphasizing durable efficacy and improvement in debilitating symptoms such as difficulty breathing, speaking, and seeing.1 3

The broadened label strategically positions Uplizna against other biologics used in gMG and related autoimmune conditions, expanding Amgen’s footprint in high-value rare disease and neuroimmunology markets.1 3 6