Nipocalimab Study Now Includes Children as Young as 2 with Myasthenia Gravis
SAN FRANCISCO, CA – November 2, 2024 - An ongoing inquiry of nipocalimab, a neonatal Fc receptor (FcRn) antagonist, has expanded to include children aged 2 to under 18 with myasthenia gravis (gMG), offering potential new treatment avenues for younger patients.Initial data presented at the 2024 American Association of Neuromuscular & Electrodiagnostic Medicine meeting indicate the drug demonstrates similar efficacy and tolerability in pediatric patients as observed in adults.
The open-label, uncontrolled multicenter analysis assesses nipocalimab’s pharmacokinetics, pharmacodynamics, safety, and activity. According to Jonathan Strober, MD, a pediatric neurologist specializing in neuromuscular disorders at the University of California, San Francisco, and Benioff Children’s Hospital, nipocalimab functions by reducing immunoglobulin levels in the body. “Basically dropping the amount of immunoglobulins you have in your body,” he explained, adding that these excess antibodies “are what cause a lot of the problems in myasthenia by attaching themselves to the receptors on the muscle.”
Data presented focused on patients aged 12 to under 18, revealing positive long-term engagement with the medication. Most patients continued treatment through 72 weeks of the safety study, with only one patient discontinuing. The drug was well-tolerated, with a low risk of adverse events, primarily consisting of common infections like those of the nose and pharynx, and COVID-19. While acknowledging a potential increased risk of infection with lowered antibody levels, Strober noted this risk “doesn’t really seem to be that significant of a risk, which is great.”
Despite promising results, recruitment for the international study remains challenging. Strober cited difficulties in identifying eligible younger patients with the necessary antibodies for enrollment and those meeting the clinical trial’s severity criteria. He also highlighted the complexities of conducting functional testing in younger cohorts.
