Abstract Details

Title Ravulizumab in Patients with Generalized Myasthenia Gravis (gMG) from Japan: Real-world Outcomes

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P7 - Poster Session 7 (5:00 PM-6:00 PM)

Poster/Presentation
Number 11-023

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.

Authors/Disclosures
Benjamin Yungher, PhD (Alexion) PRESENTER	Dr. Yungher has received personal compensation for serving as an employee of Alexion AstraZeneca Rare Disease.
Mitsuru Watanabe (Kyushu University)	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.
Kenzo Sakurai, MD	Dr. Sakurai has nothing to disclose.
Akiyuki Uzawa, MD, PhD	Dr. Uzawa has nothing to disclose.
Massanori Takahashi, MD, PhD (Clinical Neurophysiology, Osaka University)	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 as a Consultant for Alexion. 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. Takahashi has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Johnson & Johnson.
Suzuki Shigeaki, MD, PhD	Dr. Shigeaki has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx. The institution of Dr. Shigeaki has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. The institution of Dr. Shigeaki has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for alexion. Dr. Shigeaki has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for argenx. Dr. Shigeaki has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for UCB. Dr. Shigeaki has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for alexion.
Masaki Okuda, Master of Pharmacy	Mr. Okuda has received personal compensation for serving as an employee of Alexion Pharma GK. Mr. Okuda has stock in Alexion Pharmaceuticals.
Takanori Yamamoto	Mr. Yamamoto has received personal compensation for serving as an employee of Alexion Pharma Japan.
Kimiaki Utsugisawa, MD, PhD (Hanamaki General Hospital)	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.
Hiroyuki Murai, MD (International University of Health and Welfare)	Dr. Murai has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alexion. Dr. Murai has received personal compensation in the range of $500-$4,999 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.

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