The Muscular Dystrophy Association celebrates approval by the US Food and Drug Administration (FDA) to ravulizumab (Ultomiris) for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. To date, Ultomiris is the third disease modifying drug approved to treat gMG. Ultomiris will be made available in the United States (US) and marketed by Alexion, the group within AstraZeneca focused on rare diseases.
There is currently no cure for gMG. Recommended treatments for gMG include drugs that may alleviate symptoms, such as cholinesterase inhibitors, or treatments that may alter the disease course, such as immunosuppressive drugs or surgery (thymectomy). In Oct. 2017, eculizumab (marketed by Alexion as Soliris) became the first disease modifying drug approved by the FDA to treat gMG. Eculizumab was approved for adults living with the gMG subtype known as acetylcholine receptor antibody-positive (AChR ab+) gMG, which affects 80-90% of people with gMG. In Dec. 2021, efgartigimod (marketed by argenx as Vyvgart) became the second disease modifying drug approved in the US to treat adults with AChR ab+ gMG. With the current approval, adults with gMG have a new option in their therapeutic arsenal.
“The approval of Ultomiris is an important step forward in treating gMG,” said Dr. Sharon Hesterlee, Chief Research Officer for MDA. “This new drug, which was studied in 36 MDA Care Centers across the country, represents another option to meet the treatment needs of more people living with the disease and is another important milestone for the field of gMG research and drug development.”
gMG is an autoimmune disease of the neuromuscular junction. It is caused by autoreactive antibodies that destroy the communication between nerves and muscle, resulting in weakness of the skeletal muscles. Ultomiris is designed to target a component of the immune system (known as complement), which underlies many autoimmune disorders including gMG. Although treatment with Ultomiris will not cure gMG, it could lead to functional improvements that improve the daily life of individuals with gMG.