[Pubmed] Ravulizumab in Patients with Generalized Myasthenia Gravis (gMG) from Japan: Real-world Outcomes (P7-11.023)

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Neurology. 2025 Apr 8;104(7_Supplement_1):1808. doi: 10.1212/WNL.0000000000208758. Epub 2025 Apr 7.

ABSTRACT

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. Disclosure: Mitsuru Watanabe has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion Pharma, Mitsubishi Tanabe Pharma, UCB Japan. Mitsuru Watanabe has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Alexion Pharma, Argenx, UCB Japan, Novartis Pharma, Biogen Japan, Mitsubishi Tanabe Pharma, Chugai Pharmaceutical, Viatris, and Daiichi Sankyo. The institution of Mitsuru Watanabe has received research support from Japan. Mitsuru Watanabe has received publishing royalties from a publication relating to health care. Dr. Sakurai has nothing to disclose. Dr. Uzawa has nothing to disclose. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for argenx. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne Therapeutics. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Japan Clinical Research Operations. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Nippon Shinyaku. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Ono Pharmaceutical. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion Pharmaceuticals. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion Pharmaceuticals. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for argenx. Dr. Takahashi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for UCB Japan. Dr. Takahashi has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Myotonic Dystrophy Patients' Group of Japan. Dr. Shigeaki has nothing to disclose. Mr. Okuda has received personal compensation for serving as an employee of Alexion Pharma GK. Mr. Okuda has stock in Alexion Pharmaceuticals. Dr. Yungher has received personal compensation for serving as an employee of Alexion AstraZeneca Rare Disease. Mr. Yamamoto has received personal compensation for serving as an employee of Alexion Pharma Japan. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx. Dr. Utsugisawa has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB pharma. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen pharma. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Viela Bio. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Chugai. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Mitsubisi Tanabe pharma. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck. Dr. Utsugisawa has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for argenx. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Alexion phama. Dr. Utsugisawa has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Japan Blood Products Organization. Dr. Utsugisawa has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. Dr. Murai has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion Pharmaceuticals. Dr. Murai has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for argenx. Dr. Murai has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Murai has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Chugai Pharmaceutical. Dr. Murai has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Novartis. The institution of Dr. Murai has received research support from Japan Blood Products Organization. Disclaimer: Abstracts were not reviewed by Neurology® and do not reflect the views of Neurology® editors or staff.

PMID:40194082 | DOI:10.1212/WNL.0000000000208758


Source: https://pubmed.ncbi.nlm.nih.gov/4019408 ... t9+e462414