FDA Grants Priority Review to Johnson & Johnson’s Nipocalimab for Generalized Myasthenia Gravis

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The FDA has granted Priority Review to a Biologics License Application (BLA) for Johnson & Johnson’s (J&J) nipocalimab for the treatment of antibody-positive patients with generalized myasthenia gravis (gMG). According to the company, the designation was based on promising results from the Phase III Vivacity-MG3 study, which demonstrated that nipocalimab plus standard of care achieved a statistically significant reduction in Myasthenia Gravis–Activities of Daily Living (MG-ADL) scores compared to placebo, potentially improving critical functions such as eating and breathing.¹

“We welcome the FDA’s decision to grant Priority Review for the treatment of generalized myasthenia gravis, which underscores the need for additional treatment options in a broad population of people living with gMG,” said Katie Abouzahr, MD, VP, autoantibody portfolio and maternal fetal immunology disease area leader, Johnson & Johnson Innovative Medicine, in a press release. “We are committed to working closely with the FDA to help bring nipocalimab as a potential treatment to certain patients living with gMG, and we especially thank the participants in the Phase II and III studies. If approved, nipocalimab has the potential to treat gMG in antibody positive individuals, including anti-AChR, anti-MuSK, and/or anti-LRP4.”

J&J stated that the Vivacity-MG3 study was specifically designed to measure sustained efficacy and safety with consistent dosing in gMG. Investigators enrolled 199 antibody positive or negative adult gMG patients with insufficient responses to standard of care, of whom 153 were antibody positive. Patients were randomly assigned in a 1:1 ratio to receive nipocalimab plus current standard of care or placebo plus current standard of care. The primary endpoint of the study was mean change in MG-ADL score from baseline for weeks 22 through 24, with a secondary endpoint of change in QMG score. An open-label extension evaluated long-term safety and efficacy.¹

Results of the study found that patients administered nipocalimab plus standard of care achieved an improvement of 4.70 points on the MG-ADL compared to a 3.25 point improvement from baseline in the placebo plus standard of care group. Additionally, patients administered nipocalimab showed improved strength and function in different muscle groups, as measured by QMG. Further, MG-ADL response was significantly higher in the nipocalimab cohort compared to placebo at weeks 22 through 24.²

“The sustained response of nipocalimab over six months among this broad myasthenia gravis population is an important finding given the chronic, unpredictable exacerbations typically seen with myasthenia gravis,” said Carlo Antozzi, MD, neuroimmunology and muscle pathology unit of the neurological institute foundation C. Besta of Milan, Italy, in a press release. “We are encouraged by the potential of nipocalimab to uniquely help address this gap for people living with myasthenia gravis.”

According to the National Organization for Rare Disorders, gMG has a prevalence of approximately 14 to 40 per 100,000 people in the United States. The frequency of the disorder has grown over the past several decades, possibly in part from improved identification of the disease and because the frequency of autoimmune disorders has increased worldwide. Although the condition more frequently affects females, it can peak in males during their 50s or 60s. Ten percent of those infected with gMG may develop potentially life-threatening complications due to severe involvement of muscles used during breathing.³

Reference

1. Nipocalimab granted U.S. FDA Priority Review for the treatment of generalized myasthenia gravis. J&J. January 9, 2025. Accessed January 10, 2025. https://www.jnj.com/media-center/press-releases/nipocalimab-granted-u-s-fda-priority-review-for-the-treatment-of-generalized-myasthenia-gravis-gmg

2. Nipocalimab pivotal Phase 3 trial demonstrates longest sustained disease control in FcRn class. J&J. June 28, 2024. Accessed January 10, 2025. https://www.jnj.com/media-center/press-releases/nipocalimab-pivotal-phase-3-trial-demonstrates-longest-sustained-disease-control-in-fcrn-class

3. Myasthenia Gravis. NORD. Accessed January 10, 2025. https://rarediseases.org/rare-diseases/myasthenia-gravis/