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Abstract Details

Clinical Experience With Efgartigimod For Treatment Of Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis
Neuromuscular and Clinical Neurophysiology (EMG)
P1 - Poster Session 1 (8:00 AM-9:00 AM)
10-004
Efgartigimod is a first-in-class neonatal Fc receptor (FcRn) antagonist approved for treatment of acetylcholine receptor antibody-positive generalized myasthenia gravis (AChR+ gMG). In the pivotal trial, patients received an initial 4-infusion treatment cycle with subsequent treatment cycles occurring based on loss of treatment response or a minimum of 5 weeks from last infusion.
Describe experience with use of efgartigimod in clinical practice in the first year after FDA approval.
Retrospective chart review. Patients with AchR+ gMG who were treated with efgartigimod and for whom myasthenia gravis activities of daily living (MG-ADL) scores were available prior to as well as after completion of treatment cycle were included in the cohort. Patients from three major academic neuromuscular centers were included. Information regarding MG history, MG treatment(s) immediately prior to efgartigimod start, MG-ADL and other MG-specific outcome measures, laboratory data and adverse events were recorded.

Of 20 patients who received the first cycle of efgartigimod, 11 were male and 9 were female with mean age of 61.35 (+18.03) year. 14 patients (70%) had clinically meaningful improvement (> 2 points) in MG-ADL scores, whereas 3 each had no change and worsening respectively. Total IGG levels decreased by mean of 61% (± 17%) in the 8 patients for whom data was available.  

To date, over 20 patients have received at least 1 cycle of efgartigimod and of these patients, 11 have received 2 or more cycles.

Our experience suggests that at least two third of the patients with AChR+ gMG have clinically meaningful improvement in MG-ADL scores after first treatment cycle of efgartigimod. Clinical response to subsequent treatment cycles and new initiations will be discussed. 
Authors/Disclosures
Nakul Katyal, MD (University of Kentucky )
PRESENTER
Dr. Katyal has nothing to disclose.
Karen Halldorsdottir, MD Dr. Halldorsdottir has nothing to disclose.
Neelam Goyal, MD (Stanford University) Dr. Goyal has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Argenx. Dr. Goyal has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Goyal has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Janssen. An immediate family member of Dr. Goyal 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. Goyal has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alexion. The institution of Dr. Goyal has received research support from Argenx.
Raghav Govindarajan, MD, FAAN (HSHS St. Elizabeth Medical Group) Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MT pharma. Dr. Govindarajan 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. Govindarajan 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. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Catalyst. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche . Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sarepta. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Amicus. Dr. Govindarajan has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for MT pharma . Dr. Govindarajan has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Catalyst. The institution of Dr. Govindarajan has received research support from Band of Hope . The institution of Dr. Govindarajan has received research support from Alexion. Dr. Govindarajan has received publishing royalties from a publication relating to health care.
Srikanth Muppidi, MD, FAAN Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alexion. Dr. Muppidi has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for argenx. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB/Ra Pharma. Dr. Muppidi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Horizont Pharma. Dr. Muppidi has received publishing royalties from a publication relating to health care.
Ali A. Habib, MD (University of California, Irvine) Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for argenx. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Immunovant. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Dr. Habib has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Genentech/Roche. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NMD Pharma. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Alpine Immune Sciences. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NIH/NINDS. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Immunis Biomedical. Dr. Habib has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Hoffman-La Roche. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Alexion. Dr. Habib has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for argenx. The institution of Dr. Habib has received research support from Alexion. The institution of Dr. Habib has received research support from VielaBio. The institution of Dr. Habib has received research support from Immunovant. The institution of Dr. Habib has received research support from UCB. The institution of Dr. Habib has received research support from argenx. The institution of Dr. Habib has received research support from CabalettaBio. The institution of Dr. Habib has received research support from Genentech. The institution of Dr. Habib has received research support from Regeneron.