Abstract Details

Title Effectiveness and Safety of Ravulizumab in Generalized Myasthenia Gravis (gMG): Updated Analysis from a Global Registry

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P1 - Poster Session 1 (11:45 AM-12:45 PM)

Poster/Presentation
Number 11-014

Objective To assess updated effectiveness and safety data for the complement protein C5 inhibitor therapy (C5IT) ravulizumab among patients with gMG in routine clinical practice.

Background In prior analyses from the ongoing, global MG SPOTLIGHT Registry (NCT04202341), ravulizumab was well tolerated and effective in clinical practice.

Design/Methods MG-ADL total scores and MGFA classification were assessed in patients enrolled in the MG SPOTLIGHT Registry who received ravulizumab, including those who received ravulizumab only (ravu-only subgroup) or transitioned from eculizumab to ravulizumab (ecu-to-ravu subgroup) with data available prior to C5IT initiation (“pre-C5IT”) and ≥1 assessment post-ravulizumab initiation (“post-ravu”). This descriptive study had no adjustment for covariates. Safety was assessed in all patients.

Results 114/249 patients enrolled as of 1/1/2024 met inclusion criteria (male: 62%; mean±SD age at MG diagnosis: 57.9±18.6 years). Among patients with 1 pre-C5IT and 2 post-ravu assessments (n=52), average ravulizumab treatment duration was 4.1 months at first post-ravu assessment (FA) and 10.4 months at last post-ravu assessment (LA). Mean±SD MG-ADL scores improved (pre-C5IT: 7.6±3.6; FA: 3.9±3.6; LA: 3.4±3.3), as did the proportions of patients with minimal symptom expression (MSE, MG-ADL≤1) (pre-C5IT: 1/52 [2%]; FA: 18/52 [35%]; LA: 17/52 [33%]) and MGFA classification 0-II (pre-C5IT: 18/45 [40%]; FA: 41/45 [91%]; LA: 40/45 [89%]). In the ecu-to-ravu subgroup, improvements were largest post-eculizumab initiation (pre-C5IT vs last eculizumab assessment: mean±SD MG-ADL, 8.0±4.0 vs 4.4±4.2; MSE, 1/24 [4%] vs 7/24 [29%]; MGFA 0-II, 5/21 [24%] vs 19/21 [90%]) with sustained/gradual improvements through LA (3.0±2.8; 9/24 [38%]; 20/21 [95%]). In the ravu-only subgroup, outcomes improved post-ravu (pre-C5IT vs LA: mean±SD MG-ADL, 6.3±3.0 vs 4.0±3.4; MGFA 0-II, 9/14 [64%] vs 12/14 [86%]). Ravulizumab was well tolerated; no meningococcal infections were reported. Updated data will be presented.

Conclusions Consistent with prior findings, these results demonstrate the long-term effectiveness and safety of ravulizumab in patients with gMG in routine clinical practice.

Authors/Disclosures
Pushpa Narayanaswami, MD, MBBS, FAAN (Beth Israel Deaconess Medical Center) PRESENTER	Dr. Narayanaswami has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for UCB. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving as a Consultant for CVS. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Janssen. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx USA. Dr. Narayanaswami 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. Narayanaswami has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Argenx. Dr. Narayanaswami has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for NMD pharma. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for EMD-Serono (Merck usa). Dr. Narayanaswami has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Immuneabs. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Viridian. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta-Bio. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. Dr. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amgen. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Dianthus. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cartesian. Dr. Narayanaswami has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vor Bio. Dr. Narayanaswami has received personal compensation in the range of $5,000-$9,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Muscle and Nerve, Wiley. Dr. Narayanaswami has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Neurology. Dr. Narayanaswami has or had stock in Dr. Reddys laboratories.Dr. Narayanaswami has or had stock in Doximity. The institution of Dr. Narayanaswami has received research support from Alexion. The institution of Dr. Narayanaswami has received research support from NIH. The institution of Dr. Narayanaswami has received research support from Argenx. The institution of Dr. Narayanaswami has received research support from Cabaletta Bio. Dr. Narayanaswami has received publishing royalties from a publication relating to health care. Dr. Narayanaswami has a non-compensated relationship as a Member, Finance Committee with AANEM that is relevant to AAN interests or activities.
Michael T. Pulley, MD, PhD, FAAN (University of Florida Jacksonville)	Dr. Pulley has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Pulley has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MT Pharma. Dr. Pulley has received personal compensation in the range of $500-$4,999 for serving as a Consultant for amylyx. Dr. Pulley has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amgen. Dr. Pulley has received personal compensation in the range of $500-$4,999 for serving as a Consultant for csl/behring. Dr. Pulley has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cabaletta. Dr. Pulley has received personal compensation in the range of $50,000-$99,999 for serving as an Expert Witness for Multiple law firms.
Samir P. Macwan, MD	Dr. Macwan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion,Argenx,Grifols,Kabafusion, UCB. Dr. Macwan has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Abbvie,Alexion,Argenx,Grifols and UCB. The institution of Dr. Macwan has received research support from Alexion, Argenx,Immunovant, UCB.
James M. Winkley, MD	Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Winkley has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for Sanofi. Dr. Winkley has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Teva. Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche. Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. The institution of Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for J&J. Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for amgen . Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for TG therapeutics. An immediate family member of Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for BMS. Dr. Winkley has received personal compensation in the range of $100,000-$499,999 for serving on a Speakers Bureau for Teva. Dr. Winkley has received personal compensation in the range of $100,000-$499,999 for serving on a Speakers Bureau for Alexion. Dr. Winkley has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for BMS. Dr. Winkley has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for amgen . Dr. Winkley has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for UCB . Dr. Winkley has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for J&J. Dr. Winkley has received personal compensation in the range of $50,000-$99,999 for serving on a Speakers Bureau for TG therapeutics. The institution of Dr. Winkley has received research support from Noveratis. The institution of Dr. Winkley has received research support from Merck. The institution of Dr. Winkley has received research support from Sanofi. The institution of Dr. Winkley has received research support from Biogen. The institution of Dr. Winkley has received research support from Alexion.
Lida Zeinali, MD (Alexion Pharmaceuticals, Inc.)	Dr. Zeinali has received personal compensation for serving as an employee of Alexion Pharmaceuticals, AstraZeneca Rare Disease. Dr. Zeinali has stock in AstraZeneca.
Chuang Liu, PhD	Dr. Liu has received personal compensation for serving as an employee of Alexion.
Vern C. Juel, MD, FAAN (Duke University Medical Center)	Dr. Juel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. Dr. Juel has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunovant. The institution of Dr. Juel has received research support from argenx. The institution of Dr. Juel has received research support from Alexion. The institution of Dr. Juel has received research support from Janssen. The institution of Dr. Juel has received research support from NIH Rare Diseases Network.
Rup Tandan, MD, FRCP, FAAN (University of Vermont (UVM)/UVM Medical Center)	Dr. Tandan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Tandan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Apellis. Dr. Tandan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Amylyx. The institution of Dr. Tandan has received research support from Alexion. The institution of Dr. Tandan has received research support from Apellis. The institution of Dr. Tandan has received research support from Cytokinetics.
Francesco Sacca, MD, FAAN (University Federico II)	Dr. Sacca has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Sacca has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lexeo. Dr. Sacca has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Biogen. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genpharm. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Novartis. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Medpharma. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Madison Pharma. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Zai Lab. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dianthus. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Reata. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sandoz. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neopharm Israel. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Johnson&Johnson. Dr. Sacca has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Sacca has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Astrazeneca. The institution of Dr. Sacca has received research support from AIFA. The institution of Dr. Sacca has received research support from FARA.
Andrew J. Gordon, MD (Northwest Neurology)	Dr. Gordon has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Catalyst. Dr. Gordon has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen. Dr. Gordon has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avion . Dr. Gordon has received personal compensation in the range of $0-$499 for serving as a Consultant for Vertex. Dr. Gordon has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Gordon has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Lundbeck. Dr. Gordon has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Abbvie. Dr. Gordon has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for Cassiday.
James F. Howard, Jr., MD, FAAN (The University of North Carolina, Dept of Neurology, CB 7025)	Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx . Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Regeneron Pharmaceuticals. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion Pharmaceuticals. Dr. Howard has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for AMGEN (Horizon Therapeutics). Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biohaven Ltd. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for CorEvitas. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck EMD Serono. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Cartesian Therapeutics. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NMD Pharma. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for H. Lundbeck A/S. Dr. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Seismic Therapeutics. Dr. Howard 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. Howard has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vor Biopharma. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Academic CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for PeerView CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Platform Q CME. Dr. Howard has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for MJH LifeSci. Dr. Howard has or had stock in Johnson & Johnson dividends.Dr. Howard has or had stock in Pfizer dividends. An immediate family member of Dr. Howard has or had stock in GlaxoSmithKline dividends.Dr. Howard has or had stock in Bristol Myer Squbb dividends.Dr. Howard has or had stock in AbbieVie Inc. The institution of Dr. Howard has received research support from Alexion Pharmaceuticals. The institution of Dr. Howard has received research support from argenx . The institution of Dr. Howard has received research support from UCB Biosciences. The institution of Dr. Howard has received research support from NIH. The institution of Dr. Howard has received research support from Centers for Disease Control/Research Triangle Institute. The institution of Dr. Howard has received research support from Cartestian Therapeutics. The institution of Dr. Howard has received research support from NMD Pharma. The institution of Dr. Howard has received research support from Ad Scientiam. The institution of Dr. Howard has received research support from Merck EMD Serono. Dr. Howard has a non-compensated relationship as a Scientific Advisiory Board member, Committee member with Myasthenia Gravis Foundation of America that is relevant to AAN interests or activities. Dr. Howard has a non-compensated relationship as a Committee member with American Assoc Neuromuscular and Electrodiagnostic Medicine that is relevant to AAN interests or activities.

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