Phase 3 clinical trials demonstrate improvements in daily function and quality of life
[by Yu, Suin] AstraZeneca Korea announced that its C5 inhibitor, 라이브 바카라사이트 (ravulizumab), has been approved for national health insurance coverage for adult patients with generalized myasthenia gravis (MG) who test positive for anti-acetylcholine receptor (AChR) antibodies, effective December 1.
라이브 바카라사이트 gravis (MG) is a rare, chronic autoimmune disorder characterized by impaired neuromuscular transmission. Approximately 85% of patients present with autoantibodies targeting acetylcholine receptors. When these antibodies bind to the receptors, they trigger activation of the complement cascade, leading to destruction of the postsynaptic membrane. This structural damage weakens nerve-to-muscle signaling and results in compromised neuromuscular transmission.
Major symptoms include double vision, ptosis, dysphagia, shortness of breath, and mobility impairment. If not properly managed, these symptoms can progress to a potentially life-threatening condition known as a ‘myasthenic crisis.’ Accordingly, maintaining continuous control of disease activity and minimizing symptom onset are central treatment objectives. Generalized myasthenia gravis is also characterized by unpredictable and fluctuating symptoms, which can create instability in patients’ social and family lives and significantly reduce overall quality of life.
Currently, steroids and immunosuppressants serve as the primary treatments for MG. However, these therapies have limitations in efficacy and carry risks of long-term adverse effects. Ultomiris is eligible for insurance coverage for adults with MG who test positive for anti-acetylcholine receptor (AChR) antibodies and who meet one of these conditions: MGFA II to IV, MG-ADL (Myasthenia Gravis Activities of Daily Living) ≥ 6, have experienced at least one myasthenic crisis within the past year and received plasmapheresis or intravenous immunoglobulin (IVIg), or have been treated with corticosteroids and two or more non-steroidal immunosuppressants (azathioprine, cyclosporine, mycophenolate mofetil, tacrolimus, etc.) for at least three months each, but have shown refractoriness or been unable to continue due to serious adverse effects. However, coverage is not available for patients currently in myasthenic crisis or for those within 12 months of undergoing thymectomy.
"Although the recent insurance coverage for Ultomiris marks a meaningful advance in the treatment of MG, where therapeutic options were limited, the number of patients who meet the current reimbursement criteria remains extremely small. A more realistic and comprehensive relaxation of these criteria is necessary so that patients who do not respond to existing treatments can restore their quality of life and return to daily lives through access to new therapies," said Professor Shin Ha-young of the Department of Neurology at Severance Hospital.
The insurance coverage decision is based on CHAMPION-MG, a randomized, multicenter, double-blind Phase 3 clinical trial that assessed the efficacy and safety of 26 weeks of treatment in 175 adult patients with MG who tested positive for anti-acetylcholine receptor (AChR) antibodies. The primary endpoint was the MG-ADL (라이브 바카라사이트 Gravis Activities of Daily Living) score at Week 26 compared to baseline. Secondary endpoints included multiple indicators measuring quality-of-life improvement, such as the Quantitative 라이브 바카라사이트 Gravis (QMG) score, which evaluates muscle weakness.
The study demonstrated that 라이브 바카라사이트 met its primary endpoint, showing a statistically significant improvement in the total MG-ADL score, with a 3.1 point increase at Week 26 compared to a 1.4-point decline in the placebo group (P<0.001). Notably, 34.1% of patients in the placebo group achieved an improvement of at least a 3 points in the MG-ADL score, whereas more than half (56.7%) of the 라이브 바카라사이트 group reached this threshold.
The change in the QMG score, a key secondary endpoint, also showed a significant improvement, with a -2.8 point difference compared with -0.8 points in the placebo group (P<0.001). Notably, the proportion of patients achieving an improved of at least 5 points in the QMG score was more than twice as high in the 라이브 바카라사이트 group (30.0% vs. 11.3%, p=0.005).
In addition, in an open-label extension study assessing the long-term efficacy of 라이브 바카라사이트, the MG-ADL score demonstrated a mean improvement of 4 points at three years (164 weeks), and 41.8% of treatment responders achieved minimal manifestations or became essentially symptom-free.
"This reimbursement of Ultomiris is meaningful in that it offers a more stable and sustainable treatment option for patients in Korea with generalized myasthenia gravis, who have long faced limited treatment choices. We will continue working to enhance access to innovative treatments and to establish an optimal treatment environment," stated Kim Chul-woong (CW), Business Unit Director for Rare Disease at AstraZeneca Korea.