FDA

Uplizna OK’d for generalized myasthenia gravis

December 19, 2025

On December 11, 2025, FDA 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.

Efficacy

Approval of was based data from the phase 3 Myasthenia Gravis Inebilizumab Trial (MINT; NCT04524273), 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. Patients on steroids at baseline began tapering at week 4 to reach prednisone 5 mg per day by week 24. By week 26, 87.4% of patients taking Uplizna and 84.6% of those taking 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).

Safety

The most common adverse reactions in gMG patients treated with Uplizna were headache and infusion-related reactions.

Sources:

Uplizna (inebilizumab-cdon) [package insert]. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/761142s004lbl.pdf Revised December 2025. Accessed December 18, 2025.

FDA approves Uplizna® for adults with generalized myasthenia gravis. [News release]. 2025. https://www.amgen.com/newsroom/press-releases/2025/12/fda-approves-uplizna-for-adults-with-generalized-myasthenia-gravis