About Us

Our mission is to empower people living with neuromuscular diseases to live longer, more independent lives.

Muscular Dystrophy Association Celebrates FDA Approval of ZILBRYSQ® zilucoplan for the Treatment of Generalized Myasthenia Gravis (gMG) in Adult Patients

NEW YORK, NY – October 18, 2023 – The Muscular Dystrophy Association (MDA) celebrates the US Food and Drug Administration (FDA) approval of ZILBRYSQ® zilucoplan for the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody-positive. Zilucoplan is the first once-daily subcutaneous, targeted C5 complement inhibitor for gMG, and it is the only once-daily gMG-target therapy for self-administration. Zilucoplan will be made available in the United States by UCB.

Breaking News: New FDA Drug Approval for generalized Myasthenia Gravis (mMG).
Muscular Dystrophy Association celebrates the US Food and Drug Administration (FDA) approval of ZILBRYSQ® zilucoplan for the treatment of generalized myasthenia gravis (gMG) in adult patients.

"The recent approval of zilucoplan is great news for people living with gMG,” said Sharon Hesterlee, Ph.D., Chief Research Officer, MDA. “This is the second gMG therapy out of UCB that has achieved FDA approval this year, providing patients and physicians with important options. It is also the first and only self-administered therapeutic of its kind, greatly improving access, convenience, and independence for those living with and managing the disease.”

“People living with gMG can experience a variety of serious problems, including drooping eyelids, double vision, and difficulty swallowing, chewing, and talking. In some severe cases, patients may even experience life-threatening weakening of respiratory muscles,” said Barry Byrne, M.D., Ph.D., Chief Medical Advisor, MDA and Associate Chair of Pediatrics and Director of the Powell Gene Therapy Center at the University of Florida. “A particularly challenging disease to treat and manage, it is wonderful that we are now adding this new therapy to the community’s toolkit.”  

About generalized Myasthenia Gravis (gMG)

Generalized myasthenia gravis is a rare, chronic, heterogeneous, unpredictable autoimmune disease characterized by dysfunction and damage at the neuromuscular junction (NMJ).4,5,6 Several factors are understood to be drivers of gMG disease pathology, including the complement cascade, immune cells and pathogenic Immunoglobulin G (IgG) autoantibodies. gMG affects both males and females and occurs across all racial and ethnic groups. It most commonly impacts young adult females (under 40) and older males (over 60), but it can occur at any age, including childhood. It is not inherited nor is it contagious, but occasionally the disease may occur in more than one member of the same family.

Clinical Trials Support Approval of Zilucoplan

The FDA approval of zilucoplan is supported by safety and efficacy data from the RAISE study (NCT04115293), published in The Lancet Neurology in May 2023. The RAISE study was a multi-center, phase 3, randomized, double-blind, placebo-controlled study to assess the efficacy, safety profile, and tolerability of zilucoplan in adult patients with anti-acetylcholine receptor (AChR) antibody-positive gMG. Patients were randomized in a 1:1 ratio to receive daily subcutaneous (SC) injections of 0.3 mg/kg zilucoplan or placebo for 12 weeks. The study demonstrated that zilucoplan delivered rapid, consistent, and statistically significant benefits in different patient-and-clinician reported outcomes at week 12 in a broad population of adult patients with mild-to-severe anti-AChR-antibody positive gMG. The most common adverse reactions (≥10%) in patients with gMG were injection site reactions, upper respiratory tract infection, and diarrhea.

For more information about the trial, visit https://clinicaltrials.gov/ct2/show/NCT04115293.

Muscular Dystrophy Association’s Research Funding in Myasthenia Gravis

Since its inception, MDA has invested nearly $57 million in myasthenia gravis (MG). For more information, please visit MDA research.

MDA Resource Center

The neuromuscular community may contact the MDA Resource Center for guidance and support by phone at 1-833-ASK-MDA1 (1-833-275-6321) or by email at ResourceCenter@mdausa.org.

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, X, Threads, TikTok, LinkedIn, and YouTube.