U.S. FDA Grants Priority Review to Nipocalimab for Generalized Myasthenia Gravis Treatment

Johnson & Johnson has announced that the U.S. Food and Drug Administration (FDA) has awarded Priority Review status to its Biologics License Application for nipocalimab, a potential treatment for patients with generalized myasthenia gravis (gMG). This application, supported by data from the Phase 3 Vivacity-MG3 study, aims to address the needs of antibody-positive patients, including those with anti-AChR, anti-MuSK, and anti-LRP4 antibodies.

The FDA's Priority Review designation is reserved for medical applications that, if approved, would significantly enhance the treatment, diagnosis, or prevention of serious conditions compared to current therapies. Dr. Katie Abouzahr, Vice President at Johnson & Johnson, expressed excitement over this designation, highlighting the need for more treatment options for gMG patients. She emphasized the company's commitment to working with the FDA to make nipocalimab available to suitable patients, especially thanking those involved in the clinical trials.

Generalized myasthenia gravis is a chronic autoimmune disorder characterized by muscle weakness and fatigue, affecting around 700,000 individuals globally. Currently, there is no cure for gMG, which impacts the neuromuscular junction by producing antibodies that disrupt normal nerve-muscle communication. The condition can cause varied symptoms, such as limb weakness, eyelid drooping, vision issues, and difficulties in chewing, swallowing, speaking, and breathing.

The Phase 3 Vivacity-MG3 study was pivotal in evaluating the efficacy and safety of nipocalimab. In this trial, adult gMG patients who did not respond adequately to standard care were assessed. The study involved 199 participants, 153 of whom were antibody-positive. Over 24 weeks, participants received either nipocalimab or a placebo alongside their ongoing treatment. The primary outcome measured was the change in the MG-ADL score, which reflects the severity of myasthenic symptoms, in antibody-positive patients. Results showed that nipocalimab, combined with standard care, led to a significant improvement in symptoms compared to placebo.

Johnson & Johnson has also submitted a Marketing Authorisation Application to the European Medicines Agency for nipocalimab in gMG and has received Breakthrough Therapy Designation from the FDA for its potential use in treating moderate-to-severe Sjögren's disease, based on findings from another study.

Nipocalimab is a monoclonal antibody developed to inhibit the neonatal Fc receptor (FcRn), reducing the levels of circulating immunoglobulin G (IgG) antibodies. This mechanism aims to lower the impact of autoantibodies without affecting other immune functions, thus addressing conditions driven by these antibodies across various disease areas, including rare autoimmune diseases and maternal fetal conditions.

The drug has received several key designations from both the FDA and the European Medicines Agency, including Fast Track and Orphan Drug status for various conditions like hemolytic disease of the fetus and newborn, warm autoimmune hemolytic anemia, and fetal neonatal alloimmune thrombocytopenia.

Johnson & Johnson, through its subsidiaries Janssen Research & Development and Janssen Biotech, continues to focus on healthcare innovation, aspiring to create a future where complex diseases can be effectively managed and cured. The company remains committed to delivering medical breakthroughs to improve health outcomes globally.