FDA APPROVES UPLIZNA® FOR ADULTS WITH GENERALIZED MYASTHENIA GRAVIS

Core Insights - Amgen has received FDA approval for UPLIZNA (inebilizumab-cdon) as the first CD19-targeted B cell therapy for treating generalized myasthenia gravis (gMG) in adults who are positive for anti-acetylcholine receptor (AChR) and anti-muscle specific tyrosine kinase (MuSK) antibodies, offering a new treatment option with the potential for long-term disease control through biannual dosing after initial loading doses [1][7][20] Group 1: Treatment Efficacy and Approval - The approval is based on the Myasthenia Gravis Inebilizumab Trial (MINT), which is the largest Phase 3 study for gMG, demonstrating significant efficacy in reducing symptoms and steroid dependency among patients [4][5] - At Week 26, UPLIZNA showed a 1.9-point improvement in the Myasthenia Gravis Activities of Daily Living (MG-ADL) score compared to placebo, with a statistically significant p-value of <0.0001 [6][11] - The trial included 238 adults, with 87.4% of UPLIZNA patients reducing their steroid dose to 5 mg or less per day by Week 26, indicating a reduction in steroid dependency [4][14] Group 2: Patient Impact and Market Potential - UPLIZNA offers a convenient dosing schedule of twice a year, which is expected to improve patient adherence and quality of life by providing six months of treatment-free time between doses [2][7] - The prevalence of gMG in the U.S. is estimated to be between 80,000 and 100,000 patients, with a growing global incidence, highlighting a significant market opportunity for UPLIZNA [3][17] - This approval marks the third indication for UPLIZNA, previously approved for anti-AQP4 antibody positive neuromyelitis optica spectrum disorder (NMOSD) and Immunoglobulin G4-related disease (IgG4-RD), showcasing the drug's versatility [7][20] Group 3: Safety and Adverse Reactions - Common adverse reactions reported in gMG patients include headache and infusion-related reactions, with a noted incidence of 10.1% during the clinical trials [8][30] - Amgen emphasizes its commitment to patient support and access to UPLIZNA, indicating a proactive approach to managing treatment-related challenges [8][39]