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Muscular Dystrophy Association Celebrates FDA Approval of UCB’s RYSTIGGO for the Treatment of Generalized Myasthenia Gravis

• FDA approval of RYSTIGGO^® (rozanolixizumab-noli) has been granted under the Priority Review designation for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive
• Rystiggo provides an additional treatment option and opportunity for clinicians to tailor their therapeutic approach based on individual patient needs

NEW YORK, June 27, 2023 -- The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of RYSTIGGO (rozanolixizumab-noli) for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. RYSTIGGO will be made available and marketed in the United States by UCB.

RYSTIGGO is the only FDA-approved treatment in adults for both anti-AChR and anti-MuSK antibody-positive gMG, the two most common subtypes of gMG, and will be administered as an injection for subcutaneous infusion.

MG is a rare and chronic autoimmune disease where IgG autoantibodies disrupt communication between nerves and muscles, causing debilitating and potentially life-threatening muscle weakness. Approximately 85% of people with MG progress to gMG within 24 months, where muscles throughout the body may be affected. 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). Over the past six years, four therapies have been approved by the FDA to treat adults living with acetylcholine receptor antibody-positive (AChR ab+) gMG. These include eculizumab (Soliris), efgartigimod (Vyvgart), ravulizumab (Ultromiris), and recently approved efgartigimod alfa and hyaluronidase-qvfc (Vyvgart Hytrulo).

RYSTIGGO is a humanized IgG4 monoclonal antibody that binds to the neonatal Fc receptor (FcRN), resulting in the reduction of circulating immunoglobulin G (IgG). It has been designed to block the interaction of FcRn and IgG, accelerating the catabolism of antibodies and reducing the concentration of pathogenic IgG autoantibodies. Although treatment with RYSTIGGO will not cure gMG, it could lead to functional improvements that improve the daily life of individuals with gMG.

“Rystiggo is a welcome addition to the targeted therapeutic options available for the treatment of generalized myasthenia gravis,” said Sharon Hesterlee, Ph.D., Chief Research Officer, MDA. “The approval of Rystiggo will provide clinicians the opportunity to tailor their therapeutic approach based on the needs and preferences of the patient, as no two people living with gMG experience the disease the same way.”

About Generalized Myasthenia Gravis

MG is a relatively uncommon disease, with a prevalence of about 14 cases per 100,000. There are two clinical forms of MG: ocular and generalized. In ocular MG, muscle weakness often first appears in the muscles of the eyelids and other muscles that control movement of the eye (extraocular muscle). In gMG, the weakness involves the ocular muscles and a variable combination of the arm, legs, and respiratory muscles. MG can occur at any age; the data presents a peak among females in their second decade of life and older males in their sixth to

eighth decades. Today, researchers are working to understand the specifics of the autoimmune problems in MG and to refine their detection and treatment. An important area of current research is understanding the physiology of the neuromuscular junction (NMJ), with the goal of improving its function despite an immune system attack. Download MDA’s Myasthenia gravis (MG) Fact Sheet.

Clinical Trials Support Approval of RYSTIGGO

The FDA based its decision to grant approval to RYSTIGGO based on the positive results of the pivotal Phase 3 MycarinG study in gMG^, a large phase 3 study which demonstrated treatment with rozanolixizumab-noli resulted in statistically significant improvements in gMG-specific outcomes, including everyday activities such as breathing, talking, swallowing, and being able to rise from a chair.

Muscular Dystrophy Association’s Investment in gMG Research

MDA’s commitment to research on myasthenia gravis (MG) began many years ago when little was known about the cause of MG and its mortality rate was high. In the early 1970s, MDA-funded researchers helped establish the autoimmune nature of MG, demonstrating that people with the disease have a reduced number of acetylcholine (ACh) receptors and that antibodies to the receptors can induce MG in preclinical studies. These discoveries led swiftly to the lifesaving use of immunosuppressant drugs to treat the disease.

MDA-supported scientists in countries around the world are working to reveal numerous facets of MG, from identifying possible causes and triggers to understanding the disease's molecular underpinnings to developing specific treatment strategies. For more information, see Research.

About Muscular Dystrophy Association

Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and related neuromuscular diseases. For over 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 empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, Twitter, TikTok, LinkedIn, and YouTube.