James F. Howard, Jr., MD, FAAN, University of North Carolina School of Medicine, Chapel Hill, NC, discusses the potential of efgartigimod to provide an individualized treatment approach for patients with generalized myasthenia gravis (gMG). Efgartigimod, an FcR inhibitor, belongs to the second new class of drugs developed in recent years for the treatment of gMG. It was designed to be cyclically dosed based on two main factors. First, patient feedback gathered through focused group discussions indicated a preference for treatment only when necessary rather than on an ongoing basis. Second, this dosing approach aimed to evaluate the durability of the treatment response. The ADAPT trial revealed that within eight weeks, half of the patients experienced improved benefits based on trial criteria, and more than a third of them continued to see benefits for over 12 weeks. These response durations were longer than what is typically observed with intravenous immunoglobulins and plasma exchange. This emerging approach represents a step toward precision medicine, where treatment is tailored to individual patients based on their specific needs and preferences. Efgartigimod offers an option for patients who prefer a treatment strategy focused on administering medication when necessary. However, it is important to note that different patients may have different preferences for treatment approaches. Both efgartigimod and other complement inhibitors exhibit improved side effect profiles compared to standard-of-care drugs. Dissatisfaction surveys have indicated that adverse event profiles are a significant concern for patients, with high levels of dissatisfaction reported for treatments like corticosteroids. Efgartigimod, on the other hand, has a narrower side effect profile, making it more welcome among the gMG population. Another crucial aspect of efgartigimod is its rapid onset of action. Current standard-of-care therapies typically take 4-6 months, and in some cases up to 10-12 months, to show noticeable effects. In contrast, efgartigimod has shown results in as little as 1-2 weeks, which is significantly faster than existing treatment options. This rapid response is well-received by the patient population, as it provides relief and improvement in symptoms at a much faster rate. Overall, efgartigimod offers a promising avenue for individualized treatment in gMG. With its targeted dosing approach, improved side effect profile, and rapid onset of action, it addresses key concerns of patients and holds the potential to enhance the management of this complex autoimmune condition. This interview took place at the American Academy of Neurology Annual Meeting in 2023 in Boston, MA.
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