Johnson & Johnson (J&J) has announced that its anti-FcRn antibody nipocalimab has become the first investigational therapy to be granted breakthrough therapy designation (BTD) by the US Food and Drug Administration (FDA) as a treatment for adults with moderate-to-severe Sjögren’s disease (SjD).
A breakthrough therapy designation is given by the FDA to accelerate the development and regulatory review of potential new medicines for serious conditions that address a significant unmet medical need.
Estimated to impact approximately four million people globally, SjD is a chronic autoimmune disease that causes symptoms such as mucosal dryness, joint pain and fatigue.
Extraglandular manifestations are also common in SjD patients and may impact multiple organ systems, including the joints, lungs, kidneys and nervous system.
Nipocalimab is designed to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies, potentially without impact on other immune functions.
The regulator’s decision on the drug was supported by positive results from the mid-stage DAHLIAS study evaluating its effects in more than 160 adults with moderately-to-severely active primary SjD who were seropositive for anti-Ro60 and/or anti-Ro52 IgG antibodies.
Results presented earlier this year at the European Alliance of Associations for Rheumatology Congress demonstrated a statistically significant and clinically meaningful improvement in ClinESSDAI score, which measures disease activity across 11 organ systems, in nipocalimab-treated SjD patients versus placebo at 24 weeks compared to baseline.
A phase 3 trial of the candidate in SjD is currently underway, the company outlined.
Terence Rooney, vice president, rheumatology, immunology disease area leader, J&J Innovative Medicine, said: “[This] announcement marks an important step forward in the continued research and development of nipocalimab, the first investigational FcRn blocker to demonstrate positive results in a phase 2 study in adult patients with moderate-to-severe SjD.
“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 SjD.”
The announcement comes just one month after J&J shared positive results from a phase 2/3 trial of nipocalimab in patients with the rare autoimmune disease generalised myasthenia gravis.