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A neuromuscular disorder specialist, Dr Neelam Goyal shares data with efgartigimod, a neonatal Fc receptor inhibitor, for treatment of myasthenia gravis.
This is a video synopsis of a discussion involving Neelam Goyal, MD, providing insights into the recent advancements in myasthenia gravis (MG) treatment, specifically focusing on efgartigimod, an FcRn (neonatal Fc receptor) inhibitor. Dr. Goyal introduces efgartigimod as a first-in-class FcRn inhibitor approved for adult patients with AChR (acetylcholine receptor) antibody-positive generalized myasthenia gravis. She notes that the subcutaneous formulation of efgartigimod was approved in the summer of the same year.
Discussing the pivotal ADAPT trial, Dr. Goyal highlights the innovative approach where patients received 10 milligrams per kilogram IV infusions of efgartigimod weekly for four weeks, with subsequent monitoring. The study's primary endpoint was the MGADL (myasthenia gravis activity of daily living) score at the end of eight weeks, and patients returning to baseline MGADL within two points were retreated. The trial demonstrated statistically significant improvements in MGADL, as well as other scores, including the MG composite and MGQL-15 (myasthenia gravis quality of life 15).
Dr. Goyal discusses the good tolerability of efgartigimod, with common side effects being headache and upper respiratory infection, while noting additional site reactions in the subcutaneous formulation. She shares a case where she successfully used efgartigimod in a patient with AChR-positive generalized MG, achieving remission after several cycles.
Emphasizing the significance of these new therapies, Dr. Goyal expresses excitement about the enthusiasm to bring more treatments to MG patients, especially those with refractory and resistant disease. The case example underscores the positive impact of efgartigimod in achieving remission and highlights the potential of FcRn inhibitors in reshaping MG treatment paradigms.
Video synopsis is AI-generated and reviewed by NeurologyLive® editorial staff.