James Howard Jr., MD, Distinguished Professor of Neuromuscular Disease and Professor of Neurology and Medicine at UNC School of Medicine, reviews the treatment landscape for myasthenia gravis.
Myasthenia gravis is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control the eyes, eyelids, facial expressions, chewing, talking, and swallowing. The condition is usually due to the presence of antibodies against acetylcholine receptors in the neuromuscular junction.
As Dr. Howard explains, due to the variety of ways this rare disease can present, treatment of myasthenia gravis is individualized to each patient based on their specific symptoms and unique comorbidities. In addition, the financial situation of each patient is taken into account.
Acetylcholinesterase inhibitors are often used as bridge therapies. Corticosteroids are also common bridge therapies in younger patients, while steroid sparing agents are more common in older patients or in patients with contraindications to steroids. In patients under the age of 60 with generalized disease and who are anti-acetylcholine receptor (AChR) antibody positive, the removal of the thymus gland is common.
Dr. Howard goes on to discuss approved treatments for myasthenia gravis. In 2017, eculizumab, a complement inhibitor, was approved by the U.S. Food and Drug Administration (FDA) for the treatment of generalized myasthenia gravis in adult patients who are anti-acetylcholine receptor (AChR) antibody positive.
More recently, in December 2021, the FDA approved efgartigimod alfa, an FcRn inhibitor, for the treatment of this same patient population. This approval was based on positive results from the global phase 3 ADAPT trial, which were published in the July 2021 issue of The Lancet Neurology. As Dr. Howard notes, each patient within this trial demonstrated unique responses to efgartigimod, with some patients experiencing treatment effects for three or more months.
While Dr. Howard stresses the need for individualized treatment for all myasthenia gravis patients, he is optimistic about the future of FcRn inhibitors in the treatment of myasthenia gravis.
To learn more about myasthenia gravis, visit our Myasthenia Gravis Learning Center here.