Amgen announced in a press release that the US Food and Drug Administration (FDA) has approved Uplizna (inebilizumab-cdon) for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) and anti-muscle specific tyrosine kinase (MuSK) antibody positive. According to the company, the approval offers patients a new targeted treatment option that has the potential for long-term disease control with 2 doses a year, after 2 initial loading doses.

The approval of Uplizna for gMG is supported by data from the Myasthenia Gravis Inebilizumab Trial (MINT), the largest phase 3 biologic study to include both AChR+ and MuSK+ patients, and the first to successfully incorporate a steroid taper into its protocol, according to the company press release.

Patients on steroids at baseline began tapering at Week 4 to reach prednisone 5 mg per day by Week 24, according to the trial. By Week 26, 87.4% of patients taking Uplizna and 84.6% of those taking a placebo had reduced their steroid dose to 5 mg or less per day.

At Week 26, Uplizna demonstrated a 1.9-point difference in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score compared with placebo (-4.2 vs -2.2; p<0.0001). Benefits in the AChR+ patient subgroup continued through Week 52—with an exploratory analysis of AChR+ patients showing a 2.8-point difference in MG-ADL for Uplizna compared with placebo (-4.7 vs -1.9; 95% CI: −3.9 to −1.7).

According to Amgen, this is the third indication for Uplizna, which was previously approved by the FDA in June 2020 for the treatment of adult patients who have anti-aquaporin-4 (AQP4) antibody positive neuromyelitis optica spectrum disorder (NMOSD), and for the treatment of adult patients with Immunoglobulin G4-related disease (IgG4-RD) in April 2025. The most common adverse reactions in gMG were headache and infusion-related reactions.