Myasthenia Gravis and the Need for Targeted Therapies

Sindhu Ramchandren, MD, Global Clinical Leader at Janssen Pharmaceuticals, explains the pathophysiology of myasthenia gravis and the need for more targeted therapies.Myasthenia gravis is an autoimmune, neuromuscular disorder 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 presence of antibodies against acetylcholine receptors in the neuromuscular junction usually causes the condition.As Dr. Ramchandren explains, myasthenia gravis is an autoimmune disease in which the person’s own immune system attacks ACh-receptors on the neuromuscular junction. The current standard of care for myasthenia gravis is to suppress the immune system, usually with broad-acting immunosuppressants. Dr. Ramchandren believes a more targeted approach would be of greater benefit to patients. Janssen is currently developing nipocalimab, a monoclonal antibody that binds to neonatal Fc receptors (FcRn). FcRn is a protein that naturally drives IgG recycling in the body. By blocking FcRn, nipocalimab reduces the levels of IgG autoantibodies while preserving the rest of the immune system.Recently, in a phase 2 clinical trial, patients with myasthenia gravis were given nipocalimab. As a result, a dose-dependent rapid effect was observed on various disease biomarkers, including reduced titers of autoantibodies. Currently, A phase 3 clinical trial is underway (NCT04951622). To learn more about this and other autoimmune disorders, go to checkrare.com/diseases/autoimmune-auto-inflammatory-disorders/ Rare Discussions is produced by CheckRare, the leading multimedia platform dedicated to advancing education, awareness, and innovation across the rare disease community.Explore additional physician interviews, podcasts, CME activities, and rare disease resources at CheckRare.com.Subscribe to the CheckRare Podcast Network for expert conversations, weekly news, accredited education, and the latest advances across the rare disease community.Part of the CheckRare Podcast Network: Trusted conversations, news, education, and expert insights across the rare disease community.