Omar Sinno, MD, Medical Lead of Rare Disease at UCB, describes the current standard of care 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 results from a defect in the transmission of nerve impulses to muscles, which is due to the presence of antibodies against acetylcholine receptors (or other endplate receptor).
As Dr. Sinno explains, following a diagnosis of myasthenia gravis, a patient will often receive acetylcholinesterase inhibitors. This increases the amount of acetylcholine available and thus helps with muscle activation and contraction. Then these patients will often progress to a lower-dose steroid and then a higher-dose steroid. These steroids reduce the activity of the immune system and prevent antibodies from attacking the acetylcholine receptor. If the disease progresses, patients may start on immunosuppressive therapies. The downside of this course of action is that these therapies are not always efficacious. An additional course of action is rescue therapies such as intravenous immunoglobulin (IVIg) or plasma exchange (i.e., plex therapy).
Dr. Sinno points out that a common theme among these therapies is that they are all based on symptom management. No targeted therapies or cures for myasthenia gravis are available at the moment which, according to Dr. Sinno, is a problem as patients are currently left with a large treatment burden.
Currently, two targeted therapies are being investigated by UCB to treat myasthenia gravis (rozanolixizumab; zilucoplan) and are in phase 3 clinical trials (NCT03971422; NCT04115293).
To learn more about myasthenia gravis, visit our Myasthenia Gravis Learning Center here.
