Nipocalimab Receives Breakthrough Therapy Status for Sjögren’s Disease

Last November, nipocalimab received the U.S. Food & Drug Administration’s (FDA’s) breakthrough therapy designation for the treatment of adults with moderate to severe Sjögren’s disease, a chronic and debilitating autoantibody disease with high prevalence and no FDA-approved treatments.¹ The Safety and Innovation Act, which was passed on July 9, 2012, is designed to promote innovation and speed patient access to safe and effective products.² As part of the act, the breakthrough therapy designation helps the FDA assist drug makers in expediting the development and review of new treatments for which initial clinical evidence indicates substantial improvements over already available therapies for patients with serious or life-threatening diseases.

Nipocalimab is an investigational monoclonal antibody that binds with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies. The agent does this potentially without affecting other immune functions, such as autoantibodies and alloantibodies that affect multiple conditions, including rare autoantibody diseases, maternal-fetal diseases and rheumatic conditions. Additionally, the blockade of IgG binding to FcRn in the placenta may prevent transplacental transfer of maternal alloantibodies to the fetus.

The Research

Nipocalimab’s breakthrough therapy designation is supported by results from the phase 2 DAHLIAS study, which evaluated the efficacy and safety of nipocalimab for the treatment of adult patients with moderate to severe Sjögren’s disease. At EULAR 2024, the DAHLIAS study was featured in a late-breaking oral presentation (LBA0010) and demonstrated the positive results of the agent as a potential targeted therapy of Sjögren’s disease.³

In the study, participants who received 15 mg/kg of nipocalimab experienced a greater than 70% relative improvement in systemic disease activity at week 24 than those who received placebo.³ These findings support further evaluation of the treatment through a phase 3 study, which is underway.

Michele B. Kaufman, PharmD, BCGP, is a freelance medical writer based in New York City and a pharmacist at New York Presbyterian Lower Manhattan Hospital.

References

1. Nipocalimab is the first and only investigational therapy granted U.S. FDA breakthrough therapy designation for the treatment of adults living with moderate-to-severe Sjögren’s disease [news release]. Johnson & Johnson. 2024 Nov 11.
2. U.S. Food and Drug Administration Safety and Innovation Act (). U.S. Food & Drug Administration. 2018 Mar 28.
3. Gottenberg JE, Sivils K, Campbell K, et al.  Efficacy and Safety of nipocalimab, an anti-FcRn monoclonal antibody, in primary Sjogren’s disease: Results from a phase 2, multicenter, randomized, placebo-controlled, double-blind study (DAHLIAS). Ann Rheum Dis. 2024;83:240.