JnJ Nipocalimab gets USFDA Priority Review designation for generalized myasthenia gravis
Spring House: Johnson & Johnson has announced the nipocalimab Biologics License Application (BLA) has received Priority Review designation from the U.S. Food and Drug Administration (FDA) for the treatment of antibody positive (anti-AChR, anti-MuSK, anti-LRP4) patients with generalized myasthenia gravis (gMG), as supported by findings from the Phase 3 Vivacity-MG3 study.
The FDA grants Priority Review to applications for medicines that, if approved, would offer significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.
“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, M.D., Vice President, Autoantibody Portfolio and Maternal Fetal Immunology Disease Area Leader at Johnson & Johnson Innovative Medicine. “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. If approved, nipocalimab has the potential to treat gMG in antibody positive individuals, including anti-AChR, anti-MuSK, and/or anti-LRP4.”
gMG is a chronic, life-long, rare, autoantibody-driven disease, for which no cure is currently available. gMG impacts an estimated 700,000 people worldwide. In the Phase 3 study, nipocalimab plus standard of care (SOC) demonstrated a significantly greater reduction in MG-ADL response (≥2-point improvement from baseline) compared with placebo plus SOC (p=0.0213). For someone living with gMG, a 1- to 2-point change on MG-ADL may be the difference between normal eating and frequent choking on food, or shortness of breath at rest and being on a ventilator.
Johnson & Johnson also submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of nipocalimab in gMG on September 11, 2024. In addition, nipocalimab recently received U.S. FDA Breakthrough Therapy Designation for the treatment of adults with moderate-to-severe Sjögren’s disease as supported by results from the Phase 2 DAHLIAS study.
Nipocalimab is an investigational monoclonal antibody, designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies potentially without impact on other immune functions. This includes autoantibodies and alloantibodies that underlie multiple conditions across three key segments in the autoantibody space including Rare Autoantibody diseases, Maternal Fetal diseases mediated by maternal alloantibodies and Rheumatology. Blockade of IgG binding to FcRn in the placenta is also believed to limit transplacental transfer of maternal alloantibodies to the fetus.
The FDA and European Medicines Agency (EMA) have granted several key designations to nipocalimab including:
- U.S. FDA Fast Track designation in hemolytic disease of the fetus and newborn (HDFN) and warm autoimmune hemolytic anemia (wAIHA) in July 2019, gMG in December 2021 and fetal neonatal alloimmune thrombocytopenia (FNAIT) in March 2024
- U.S. FDA Orphan drug status for wAIHA in December 2019, HDFN in June 2020, gMG in February 2021, chronic inflammatory demyelinating polyneuropathy (CIDP) in October 2021 and FNAIT in December 2023
- U.S. FDA Breakthrough Therapy designation for HDFN in February 2024 and for Sjögren’s disease in November 2024
- U.S. FDA granted Priority Review in gMG in Q4 2024
- EU EMA Orphan medicinal product designation for HDFN in October 2019
