FDA grants nipocalimab priority review for the treatment of gMG
Phase 3 VIVACITY-MG3 trial data showed therapy helped reduce disease severity
The U.S. Food and Drug Administration (FDA) has granted priority review to Johnson & Johnson’s request to approve nipocalimab for patients with generalized myasthenia gravis (gMG) who are positive for the most common types of MG-causing self-reactive antibodies.
This means the FDA intends to take action on the company’s biologics license application within six months rather than the 10 months in a standard review. Priority review is granted to therapies that, if approved, should significantly improve the safety and effectiveness at treating, diagnosing, or preventing serious conditions over standard approaches.
“We are pleased with the FDA’s decision to grant nipocalimab priority review for the treatment of [gMG]. This milestone highlights the unmet need that continues to exist for the 700,000 people worldwide living with gMG,” said Katie Abouzahr, MD, vice president and leader of the autoantibody portfolio and maternal-fetal immunology disease area at Johnson & Johnson Innovative Medicine, in an email to Myasthenia Gravis News.
“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 2 and 3 studies,” Abouzahr said in a company press release.
MG occurs when self-reactive antibodies target and attack important proteins at the neuromuscular junction, the site of communication between muscles and nerve cells, leading to symptoms of muscle weakness and fatigue.
In about 85% of patients, these self-reactive antibodies target acetylcholine receptors (AChRs), which are essential for muscle contraction. A smaller percentage of patients have self-reactive antibodies that target other neuromuscular junction proteins, including muscle-specific kinase (MuSK) and low-density lipoprotein receptor-related protein 4 (LRP4).
How does nipocalimab work in gMG?
Nipocalimab is designed to block the activity of the neonatal Fc receptor, a protein that helps prevent antibodies circulating in the bloodstream from being broken down, including those driving MG. By promoting antibody degradation, the therapy should reduce levels of the harmful self-reactive antibodies and ease MG disease severity.
The decision to grant priority review to nipocalimab’s application was supported by data from the Phase 3 VIVACITY-MG3 trial (NCT04951622), which showed the treatment led to significant reductions in MG severity.
The 24-week placebo-controlled trial enrolled 199 adults with gMG who had an insufficient response to standard therapy; 153 of them were positive for self-reactive antibodies. The participants were randomly assigned to receive infusions into a vein, or intravenously, of nipocalimab every two weeks along with standard of care, or a placebo plus standard of care.
The study met its main goal of achieving a significant easing of disease severity with nipocalimab, as measured by changes in the score of the MG Activities of Living (MG-ADL) scale. Patients treated with nipocalimab plus standard of care had a significantly greater improvement in MG-ADL scores relative to the beginning of the trial over the placebo-treated participants.
Also, significantly more nipocalimab-treated patients achieved a clinically meaningful response, defined as a minimum 2-point improvement in MG-ADL scores, relative to placebo-treated patients in the final weeks of treatment.
“Nipocalimab has the potential to offer sustained disease control in antibody positive individuals, including anti-AChR, anti-MuSK, or anti-LRP4,” Abouzahr said. “We are committed to working to advance nipocalimab as a treatment option for this devastating disease.”
Johnson & Johnson submitted a marketing authorization application last year to the European Medicines Agency seeking to treat gMG with nipocalimab in the European Union.
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