MDA Celebrates FDA Approval of Alexion’s Ultomiris for the Treatment of gMG
NEW YORK, April 29, 2022 /PRNewswire/ — The Muscular Dystrophy Association Celebrates Yesterday’s U.S. Food and Drug Administration (FDA) Approval of Ravulizumab (Ultomiris) for the Treatment of Generalized Disease myasthenia gravis (gMG) in adults who test positive for anti-acetylcholine receptor (AChR) antibody. To date, Ultomiris is the third disease-modifying drug approved to treat gMG. Ultomiris will be available in United States (USA) 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 can reduce symptoms, such as cholinesterase inhibitors, or treatments that can alter the course of the disease, such as immunosuppressive drugs or surgery (thymectomy). In October 2017, eculizumab (marketed by Alexion as Soliris) became the first FDA-approved disease-modifying drug to treat gMG. Eculizumab has been approved for adults living with the subtype of gMG known as anti-acetylcholine receptor antibody (AChR ab+) gMG, which affects 80-90% of people with gMG. In Dec. 2021, efgartigimod (marketed by argenx as Vyvgart) has become the second disease-modifying drug approved in the United States to treat adults with ab+ gMG AChR. With current approval, adults with gMG have a new option in their treatment arsenal.
“The approval of Ultomiris is an important milestone in the treatment of gMG,” said Dr. Sharon Hesterlee, director of research for MDA. “This new drug, which has been studied at 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 step in 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 communication between nerves and muscles, resulting in skeletal muscle weakness. Ultomiris is designed to target a component of the immune system (known as complement), which underlies many autoimmune diseases, including gMG. Although treatment with Ultomiris does not cure gMG, it may lead to functional improvements that improve daily life for people with gMG.
Clinical Trials Support Ultomiris Approval
The FDA based its decision to grant approval for ultomiris on positive results from the Phase 3 CHAMPION-MG trial (NCT03920293), which evaluated the safety and efficacy of Ultomiris in 175 adults with gMG living in North America, Europe, Asia Pacific and Japan.
Ultomiris was generally well tolerated by trial participants, with the most common side effects being upper respiratory tract infection and diarrhea. The trial met its primary endpoint, demonstrating that Ultomiris treatment of people with gMG (AchR-Ac+) resulted in clinically meaningful improvements in symptom severity, as measured by the Myasthenia Gravis Score Activities of Daily Living (MG-ADL). Improvements in secondary endpoints assessing disease-related and quality-of-life measures were also observed. Data presented by the trial sponsors at the 2022 American Academy of Neurology Annual Meeting demonstrated that Ultomiris has some advantages over existing treatments; treatment every eight weeks shows sustained clinical benefit over 60 weeks of treatment, compared to treatment with Soliris every two weeks. After completing the CHAMPION-MG trial, all participants were eligible to receive Ultomiris in an open-label extension period of the study, which is ongoing.
Ultomiris works by inhibiting the complement protein C5, which is a component of the body’s immune system. In the context of gMG, the uncontrolled activation of C5 and related complement proteins can cause the immune system to attack healthy cells in the body. After an initial loading dose, Ultomiris is given intravenously (into the vein) every eight weeks in adult patients. Ultomiris is also being evaluated for the treatment of people with other hematological and neurological disorders.
About the Muscular Dystrophy Association
Association of muscular dystrophy (MDA) is the first voluntary health organization in United States for people living with muscular dystrophy, ALS and related neuromuscular diseases. For more than 70 years, MDA has led the way in accelerating research, advancing care and advocating for the support of our families. MDA’s mission is to enable the people we serve to live longer, more independent lives. To find out more visit mda.org and follow MDA on instagram, Facebook, Twitter, ICT Tacand LinkedIn.
SOURCE Muscular Dystrophy Association