Nipocalimab Granted FDA Breakthrough Therapy Designation for Sjögren’s Disease
Key Insights
Johnson & Johnson's nipocalimab receives Breakthrough Therapy Designation (BTD) from the FDA for moderate-to-severe Sjögren’s Disease (search) (SjD).
The BTD is supported by positive Phase II DAHLIAS trial results, marking nipocalimab as the first FcRn (search) blocker with such outcomes in SjD.
Nipocalimab, a monoclonal antibody, targets FcRn (search) to reduce circulating IgG antibodies without broadly affecting the immune system.
Johnson & Johnson's nipocalimab has been granted Breakthrough Therapy Designation (BTD) by the FDA for the treatment of moderate-to-severe Sjögren’s Disease (search) (SjD). This makes nipocalimab the first investigational therapy to receive this designation for SjD, highlighting its potential to address a significant unmet need.
The BTD is based on data from the Phase II DAHLIAS study, a randomized, placebo-controlled, multicenter, double-blind trial. The study demonstrated positive outcomes for nipocalimab, an investigational neonatal crystallisable fragment receptor (FcRn (search)) blocker, as a potential targeted treatment for SjD. The results have paved the way for an ongoing Phase III study further evaluating the efficacy and safety of nipocalimab in patients with moderate-to-severe SjD.
Mechanism of Action
Nipocalimab is a monoclonal antibody that blocks FcRn (search) with high affinity. This action potentially reduces levels of circulating immunoglobulin G (IgG) antibodies without impacting other immune functions. This targeted approach is particularly relevant in Sjögren’s Disease (search), where autoantibodies play a key role in the disease's pathology.
Clinical Significance
Sjögren’s Disease (search) is a chronic and debilitating autoantibody disease that significantly impacts daily life and overall quality of life. It primarily affects glands responsible for saliva and tear production, leading to dryness and discomfort. The disease can also cause systemic symptoms affecting multiple organs, and patients face a potentially higher risk of developing B-cell lymphomas.
Terence Rooney, Vice President at J&J Innovative Medicine Rheumatology and Immunology Disease Area, stated, "Today’s announcement marks an important step forward in the continued research and development of nipocalimab... With no treatments currently approved that may directly address the underlying cause(s) of the disease, innovation is critically needed to improve patient outcomes in Sjögren’s disease (search)."
Prior Regulatory Designations
Nipocalimab has received several prior regulatory designations for various indications. These include a BTD in February 2024 for treating alloimmunized pregnant people at high risk of severe hemolytic disease of the fetus and newborn (HDFN), as well as fast-track status for HDFN, warm autoimmune hemolytic anemia, generalized myasthenia gravis (search) and fetal neonatal alloimmune thrombocytopenia (search).