Jonathan Strober, MD, Director of the Neuromuscular Clinic and Professor of Pediatrics and Neurology at the University of California at San Francisco, discusses the implications of nipocalimab’s approval to treat pediatric patients with myasthenia gravis (MG).
MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles. Common symptoms include weakness of the muscles that control the eye and eyelid, facial expressions, chewing, talking, and swallowing. Weakness tends to increase during periods of activity and improve after periods of rest. The condition results from a defect in the transmission of nerve impulses to muscles, which is due to the presence of antibodies against acetylcholine. The exact reason this occurs is not known.
The U.S. Food and Drug Administration (FDA) recently approved Imaavy (nipocalimab) for the treatment of generalized MG in patients ages 12 years and older who are anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) antibody positive. Nipocalimab is a human monoclonal antibody FcRn-inhibitor designed to reduce immunoglobulin G (IgG) autoantibodies.
Vivacity-MG3 Clinical Trial
The approval was supported by data from the Vivacity-MG3 clinical trial (NCT04951622). The study illustrated superior disease control through 24 weeks, measured by MG Activities of Daily Living score, in patients treated with nipocalimab plus standard of care versus those treated with placebo plus standard of care. A rapid and sustained reduction in autoantibody levels by up to 75% from the first dose and throughout a 24-week period of monitoring was also observed. Additionally, improvements in patients treated with nipocalimab plus standard of care were maintained out to 20 months of follow-up in the open-label extension.
In addition to the above study, nipocalimab is also being tested in patients as young as 2 years of age. Results from the ongoing Vibrance phase 2/3 pediatric study (NCT05265273), patients aged 2 to 17 years, showed that nipocalimab plus standard of care met its primary endpoint with a 69% reduction in total serum IgG over 24 weeks. Improvements were also seen in MG Activities of Daily Living and Quantitative MG scores. The safety profile of nipocalimab has been favorable and consistent across both studies.
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To learn more about MG and other rare neurological disorders, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/