FDA Grants Breakthrough Status to J&J's Nipocalimab for Sjögren's Disease

Johnson & Johnson (J&J) has announced that its investigational anti-FcRn antibody, nipocalimab, has been granted breakthrough therapy designation (BTD) by the US Food and Drug Administration (FDA) for treating adults with moderate-to-severe Sjögren’s disease (SjD). This designation is aimed at expediting the development and regulatory review of new medications for serious conditions that address significant unmet medical needs.

Sjögren’s disease affects roughly four million individuals worldwide and is a chronic autoimmune disorder characterized by symptoms such as dryness of mucosal surfaces, joint pain, and fatigue. The disease can also manifest extraglandularly, impacting multiple organ systems including joints, lungs, kidneys, and the nervous system.

Nipocalimab works by blocking the neonatal Fc receptor (FcRn) to reduce the levels of circulating immunoglobulin G (IgG) antibodies, which may help alleviate the symptoms of SjD without affecting other immune functions. The FDA’s decision to grant BTD to nipocalimab was influenced by positive outcomes from the DAHLIAS study. This mid-phase clinical trial evaluated more than 160 adults with moderately-to-severely active primary SjD who were seropositive for anti-Ro60 and/or anti-Ro52 IgG antibodies.

The results of this study were presented earlier in the year at the European Alliance of Associations for Rheumatology Congress. They demonstrated that patients treated with nipocalimab showed a statistically significant and clinically meaningful improvement in their ClinESSDAI scores—a measure of disease activity across 11 organ systems—compared to those who received a placebo over a 24-week period.

J&J has also indicated that a phase 3 trial of nipocalimab for SjD is currently in progress. Terence Rooney, Vice President and Rheumatology and Immunology Disease Area Leader at J&J Innovative Medicine, commented on the breakthrough designation, emphasizing the significance of this development in advancing nipocalimab. He highlighted it as the first investigational FcRn blocker to show positive results in a phase 2 study for adults with moderate-to-severe SjD. Rooney also underscored the urgent need for innovative treatments to improve patient outcomes in SjD, given the lack of approved therapies that directly address the disease's underlying causes.

The announcement follows closely on the heels of J&J’s recent disclosure of favorable phase 2/3 trial results for nipocalimab in patients with generalized myasthenia gravis, another rare autoimmune disorder.