Johnson & Johnson Submits BLA to FDA for Nipocalimab, a First-in-Class FcRn-Targeted Antibody Therapy, Seeking Global First Approval for Generalized Myasthenia Gravis

Sep 01, 2024 20:00 CST Updated 20:00

Johnson & Johnson

Healthcare Product Manufacturers, Health Service Providers

On August 29, 2024, Johnson & Johnson announced that it had submitted a Biologics License Application (BLA) to the U.S. FDA, seeking the world's first approval for its investigational neonatal Fc receptor (FcRn)-targeted antibody therapy Nipocalimab for the treatment of generalized myasthenia gravis.（Generalized masthenia gravis，gMG）。Due toNipocalimabHas the potential to treat a variety of autoimmune diseases,This therapy was highlighted by the industry media Evalua early this year.te is listed asTop 10 Potential Blockbuster TherapiesOne of。

Myasthenia gravis is an autoimmune neuromuscular disease.，Destruction of the postsynaptic membrane at the neuromuscular junction by autoantibodies leads to impaired neuromuscular transmission, clinically manifested as fluctuating skeletal muscle weakness, fatigability, worsening with activity, and improvement with rest.Symptoms are mostly distributed in the eyes, bulb, and limb muscles, and severe cases can affect the respiratory muscles, leading to respiratory failure.

This submission is primarily based on data from the Vivacity-MG3 Phase 3 clinical trial. This double-blind, placebo-controlled study enrolled patients with generalized anti-AChR+, anti-MuSK+, and/or anti-LRP4+ antibodies, which account for approximately 95% of the gMG patient population. Analysis showed that the trial met its primary endpoint, demonstrating improvement compared to baseline at weeks 22, 23, and 24.Patients receiving nipocalimab in combination with SOC treatment showed an average improvement of 4.70 points in MG-ADL scores., significantly higher than the 3.25 points in patients treated with placebo plus SOC (P=0.002).For gMG patients, a change of 1 to 2 points in the MG-ADL score may mean the difference between normal eating and frequent swallowing difficulties, or between shortness of breath at rest and the need for a ventilator.

In addition to achieving this primary endpoint, the trial also met key secondary endpoints:

In Week 22 and Week 24,Quantitative Myasthenia Gravis (QMG) score measurements show,Compared with placebo combined with SOC,Nipocalimab combined with SOC showed significantly greater improvements in strength and function across different muscle groups (P<0.001).
At weeks 22, 23, and 24, nipocalimab combined with SOC showed a significantly higher MG-ADL response (≥2-point improvement from baseline) compared to placebo combined with SOC (P=0.021), further highlighting the potential of nipocalimab treatment in reducing the impact on daily life for patients with gMG.
In the trialNipocalimabThe safety and tolerability were consistent with other trials. The overall incidence of adverse events, serious adverse events, and adverse events leading to discontinuation was similar to the placebo combined with SOC group.

Nipocalimab is a potential "best-in-class" antibody therapy targeting the neonatal Fc receptor. By binding to FcRn, it prevents autoantibodies taken up by monocytes and endothelial cells from being released back into the bloodstream, instead degrading them within the cells.Research has shown that it can reduce IgG levels, including pathogenic autoantibodies in the bloodstream, by more than 75%. This antibody therapy has the potential to treat various autoimmune antibody-mediated immune diseases. The FDA has granted this therapy Breakthrough Therapy Designation for treating pregnant women at high risk of severe Hemolytic Disease of the Fetus and Newborn (HDFN), as well as Fast Track designation for treating HDFN, gMG, warm antibody autoimmune hemolytic anemia (wAIHA), and fetal/neonatal alloimmune thrombocytopenia (FNAIT).

Source | Guangzhou Rare Disease Gene Therapy Alliance

Editor | Subi

Rare Neurological Disorders Communication Group

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