FDA Grants Breakthrough Therapy Designation to Nipocalimab for Moderate-to-Severe Sjogren's Disease in Adults
The investigational monoclonal antibody nipocalimab has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration for treating moderate-to-severe Sjögren's disease (SjD) in adults.
The latest BTD for nipocalimab is based on data from the Phase 2 DAHLIAS study, which demonstrated significant clinical benefits. Study participants receiving 15 mg/kg of nipocalimab showed, on average, more than a 70% relative improvement in systemic disease activity at Week 24 compared to the placebo group. Sjögren's disease, a chronic autoantibody-driven condition, currently lacks advanced approved therapies, making this progress particularly significant.
The DAHLIAS study evaluated the safety and efficacy of nipocalimab in adults with moderate-to-severe primary Sjögren's disease. Participants were randomised to receive varying doses of the drug or a placebo every two weeks for 22 weeks, alongside standard care. The primary endpoint measured changes in the ClinESSDAI score, a tool assessing systemic disease activity, while secondary endpoints included assessments of multiple organ systems, physician evaluations, and patient-reported outcomes.
Suffering from Sjogren’s disease can severely impact the quality of life due to symptoms like chronic dryness, fatigue, and joint pain. The disease can also lead to extraglandular complications affecting multiple organ systems and an elevated risk of developing B-cell lymphomas. Severe forms of SjD are associated with higher mortality rates, further underscoring the need for effective therapies.
Nipocalimab works by blocking the neonatal Fc receptor (FcRn) to reduce levels of circulating immunoglobulin G (IgG) antibodies, including autoantibodies that contribute to disease activity. This mechanism of action is being explored across several conditions involving autoantibodies and alloantibodies.
This development follows previous regulatory milestones for nipocalimab, including U.S. FDA Fast Track and Orphan Drug designations for conditions such as warm autoimmune haemolytic anaemia (wAIHA), generalised myasthenia gravis (gMG), and foetal and neonatal alloimmune thrombocytopenia (FNAIT). The drug has also received Orphan Medicinal Product status from the European Medicines Agency (EMA).
The Phase 2 DAHLIAS study featured late-breaking findings presented at the European Alliance of Associations for Rheumatology (EULAR) 2024 Congress.
These results are expected to support ongoing Phase 3 research into nipocalimab's potential as a targeted therapy for Sjögren's disease, offering hope to patients living with this challenging condition.
