FDA grants breakthrough status to J&J’s nipocalimab for SjD treatment
The US Food and Drug Administration (FDA) has awarded the second breakthrough therapy designation (BTD) to Johnson & Johnson‘s (J&J) nipocalimab for treating individuals with moderate-to-severe Sjögren’s disease (SjD). This marks nipocalimab as the only investigational therapy to receive this designation for SjD.
Nipocalimab’s latest BTD is underpinned by data from the Phase II DAHLIAS study, which is a randomised, placebo-controlled, multicentre, and double-blind trial. The data from the Phase II DAHLIAS study showcased the “first-ever positive” outcomes of an investigational neonatal crystallisable fragment receptor (FcRn) blocker as a potential targeted treatment of SjD.
An investigational monoclonal antibody, nipocalimab blocks FcRn with high affinity, potentially reducing levels of circulating immunoglobulin G antibodies without impacting other immune functions.
SjD is a chronic and debilitating autoantibody disease that can severely impact daily life and overall quality of life. It frequently affects glands responsible for saliva and tear production and can also cause systemic symptoms that affect multiple organs. As compared to general population, SjD patients are at a potentially higher risk of developing B-cell lymphomas.
The initial BTD for nipocalimab was granted in February 2024 for treating alloimmunised pregnant people at high risk of severe haemolytic disease of the foetus and newborn (HDFN).
The therapy received several regulatory designations for various indications, including the US FDA’s fast-track status in July 2019 for the treatment of HDFN and warm autoimmune haemolytic anaemia; in December 2021 for treating generalised myasthenia gravis; and in March this year for foetal neonatal alloimmune thrombocytopenia treatment.
J&J Innovative Medicine Rheumatology and Immunology Disease Area leader and vice-president Terence Rooney said: “Today’s 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 II study in adult patients with moderate-to-severe Sjögren’s disease. 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.”
