The director of the Pediatric Neuromuscular Program at UCSF Benioff Children’s Hospital San Francisco discussed 2-year findings from the phase 2/3 VIBRANCE-MG study of nipocalimab in adolescents with generalized MG. [WATCH TIME: 6 minutes]
Juvenile myasthenia gravis (MG) is rare. A US claims-based analysis estimated a 2020 incidence of 5.9 to 8.7 per million person-years, with consistently higher rates among girls than boys, yet most treatments used in this population have been approved only for adults.1 For decades, management of
These agents are effective but often limited by delayed onset, long-term toxicity, and incomplete disease control, and evidence for specific patient populations remains limited even as complement inhibitors and neonatal Fc receptor (FcRn) inhibitors have expanded the treatment landscape.2 Nipocalimab (Imaavy; Johnson & Johnson), an FcRn blocker that lowers circulating IgG, was approved for adolescents 12 years and older with gMG in part through pharmacokinetic and pharmacodynamic extrapolation from adult data.3
In the pivotal phase 3 VIVACITY-MG3 trial (NCT04951622) in adults, nipocalimab added to standard of care improved Myasthenia Gravis Activities of Daily Living (MG-ADL) scores by 4.70 points, compared with 3.25 points with placebo, over weeks 22 to 24.4 The open-label phase 2/3 VIBRANCE-MG study (NCT05265273) previously showed
At the
WATCH TIME: 6 minutes | Captions are auto-generated and may contain errors.
“As a pediatric neurologist, we’ve always used drugs that were approved for adults, but never tested in children.”
To discuss these findings, NeurologyLive® spoke with
He explained how FcRn inhibition prevents antibody recycling and noted that IgG reductions were rapid and robust. Some participants developed infections during the trial, and 1 required a temporary pause in treatment before resuming. Strober also emphasized that improvements on both the MG-ADL and QMG were sustained without fluctuation through 2 years, which he said is important because many of these patients will carry the disease into adulthood.
From there, Strober outlined the key clinical takeaways. He identified safety as the top priority in a growing population, followed by efficacy and the ability to reduce corticosteroids, which he described as a common first-line treatment that carries bone-related and other long-term risks. He then discussed next steps, including the value of longer follow-up for detecting late safety signals in a field where many new neuromuscular therapies lack long-term data.
He also stressed the need to enroll children younger than 12 years, noting that insurers often follow the label and that off-label access is difficult. Strober closed by highlighting that adolescent MG is often overlooked and that dedicated pediatric trials let clinicians treat these patients with greater confidence.
