On March 13, Amgen announced itsRare Disease Drug Uplizna (inebilizumab-cdon) in the Treatment of Myasthenia Gravis (MG)Latest Phase III Clinical Trial Data: 52-week results of Uplizna show,Uplizna Expected to Become the New Standard for Myasthenia Gravis Treatment, Offering Long-Lasting Symptom Relief, and simplify the treatment regimen. It is worth mentioning that a regimen requiring only two doses per year is a significant advancement for patients.
These data further solidify Uplizna's potential in the treatment of myasthenia gravis, andProvided strong support for its upcoming FDA application.
Uplizna is a humanized monoclonal antibody with high affinity for CD19.CD19 is expressed on various types of B cells, including antibody-secreting plasmablasts and plasma cells.By binding to the CD19 antigen, Uplizna can quickly eliminate these cells from the blood circulation, thereby reducing the production of autoantibodies and alleviating patient symptoms.UpliznaOriginally developed by Viela Bio,Approved by the FDA for the first time in 2020 to treat patients with neuromyelitis optica spectrum disorder (NMOSD).
The phase III clinical trial results released this time show that Uplizna is positive for acetylcholine receptor antibodies.(AChR+) myasthenia gravis patients showed significant efficacy and durability. Detailed results are as follows:During the 52-week follow-up, patients in the Uplizna groupMyasthenia Gravis Activities of Daily Living Score (MG-ADL) decreased by an average of 2.8 points from baseline., significantly better than the placebo group (95% confidence interval: -3.9 to -1.7). In addition,72.3% of patients in the Uplizna group had at least a 3-point improvement in MG-ADL scores, compared to only 45.2% in the placebo group.。In terms of the Quantitative Myasthenia Gravis Score (QMG),Patients in the Uplizna group showed greater improvement at week 52 compared to the placebo group, with an adjusted difference of -4.3 points.(95% confidence interval: -5.9 to -2.8).69.2% of patients in the Uplizna group had at least a 3-point improvement in QMG score., while the placebo group was only 41.8%.In addition, the safety of Uplizna is consistent with the known approved indications, and no new safety signals were identified.Currently,UpliznaCurrently under FDA Priority Review,For the treatment of Immunoglobulin G4-Related Disease (IgG4-RD), with an expected review date of April 3, 2025.。FDA Grants Orphan Drug Designation to Inebilizumab for gMG.The regulatory submission is currently underway and is expected to be completed in the first half of 2025.The results will be presented as a late-breaking oral presentation at the American Academy of Neurology (AAN) Annual Meeting held in San Diego on April 8, 2025.
Generalized Myasthenia Gravis (gMG) is a rare, chronic, B-cell mediated autoimmune disease that impairs neuromuscular communication and can lead to muscle weakness, breathing difficulties, swallowing problems, and speech and vision impairments.Approximately 85% of patients with myasthenia gravis have the generalized form, known as gMG.The prevalence and incidence of gMG are increasing globally. There are 80,000 to 100,000 patients with myasthenia gravis in the United States. Approximately 85% of patients with myasthenia gravis test positive for anti-AChR antibodies, and about 7% test positive for anti-MuSK antibodies. The global prevalence is estimated at 2 to 36 cases per 100,000 people. The disease is more common in young women (ages 20-30) and men aged 50 and above.
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