Ravulizumab in Patients with Generalized Myasthenia Gravis (gMG) from Japan: Real-world Outcomes
Mitsuru Watanabe1, Kenzo Sakurai2, Akiyuki Uzawa3, Masanori Takahashi4, Shigeaki Suzuki5, Masaki Okuda6, Benjamin Yungher7, Takanori Yamamoto6, Kimiaki Utsugisawa8, Hiroyuki Murai9
1Kyushu University, 2St. Marianna University School of Medicine, 3Chiba University, 4Osaka University Graduate School of Medicine, 5Keio University School of Medicine, 6Alexion Pharma GK, 7Alexion, AstraZeneca Rare Disease, 8Hanamaki General Hospital, 9International University of Health and Welfare
Objective:
The ravulizumab postmarketing surveillance (PMS) is ongoing to assess its real-world safety and effectiveness in adult patients with gMG in Japan, in accordance with regional guidelines. Here we report the first interim analysis from the ravulizumab gMG PMS.
Background:
Ravulizumab, a terminal complement inhibitor administered intravenously every 2 months, was approved for gMG in August 2022 in Japan.
Design/Methods:
These PMS data reflect mandatory all-case monitoring through December 2023. Patient demographics, Myasthenia Gravis Foundation of America Postintervention Status (MGFA-PIS), Myasthenia Gravis Activities of Daily Living (MG-ADL) scores, and safety information were reported.
Results:
The analysis sets included 49 patients (safety) and 45 patients (effectiveness), all of whom provided consent for data publication. In the effectiveness analysis set, 24 (53.3%) patients were female, and 38 (84.4%) patients had treatment history with eculizumab, a terminal complement inhibitor, during the 12 months prior to initiating ravulizumab. At ravulizumab initiation, mean age was 56.9 (SD±11.7) years old, baseline MG-ADL total score was 3.9 (SD±3.20), and average gMG duration was 132.3 (SD±127.0) months. Mean treatment duration of ravulizumab was 22.2 (SD±7.4) weeks. Regarding MGFA-PIS, 46.2% of patients reached pharmacologic remission with minimal manifestations, 20.5% were improved, and 33.3% were unchanged at 26 weeks. Zero patients were classified as worse. Mean change in MG-ADL score was −0.4 (SD±1.36). In the safety analysis set, adverse drug reactions (ADRs) were reported in 10 (20.4%) patients; six (12.2%) patients had ≥1 serious ADR. The most common serious ADRs were myasthenia gravis (n=4) and COVID-19 pneumonia (n=2). There were no meningococcal infections reported.
Conclusions:
This is the first report of the ravulizumab PMS in patients with gMG. Effectiveness benefits for patients who received prior eculizumab treatment were maintained after ravulizumab treatment, although a small number of patients was assessed at week 26. No new safety signals were detected.
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