USFDA grants Breakthrough Therapy Designation to Nipocalimab for treatment of adults with moderate-to-severe Sjögren's disease: JnJ
Spring House: Johnson & Johnson has announced that the U.S. Food and Drug Administration (FDA) has granted nipocalimab Breakthrough Therapy designation (BTD) for the treatment of adults living with moderate-to-severe Sjögren’s disease (SjD), a debilitating and chronic autoantibody disease with high prevalence, for which no approved advanced treatments are available. Nipocalimab is a investigational therapy to secure this designation in SjD. This regulatory milestone is the second time BTD has been granted for nipocalimab; the first was granted in February for the treatment of alloimmunized pregnant individuals at high risk of severe hemolytic disease of the fetus and newborn (HDFN).
“The 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 Sjögren’s disease,” said Terence Rooney, Vice President, Rheumatology, Immunology Disease Area Leader, Johnson & Johnson Innovative Medicine. “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. This milestone underscores our unwavering commitment to develop novel, transformational therapies that may help address significant unmet need for patients living with autoantibody-driven diseases.”
The BTD is supported by data from the Phase 2 DAHLIAS study evaluating the efficacy and safety of nipocalimab for the treatment of adult patients with moderate-to-severe SjD and supports further evaluation of the investigational treatment through a Phase 3 study, which is underway. Data from the nipocalimab Phase 2 DAHLIAS study were featured in a late-breaking oral presentation (LBA0010) at the European Alliance of Associations for Rheumatology (EULAR) 2024 Congress and demonstrated the first-ever positive results of an investigational FcRn blocker as a potential targeted therapy in SjD.
The FDA grants BTD to expedite the development and regulatory review of an investigational medicine that is intended to treat a serious or life-threatening condition and is based on preliminary clinical evidence that indicates the drug may have substantial improvement in at least one clinically significant endpoint over available therapy. Many patients living with SjD experience symptoms that interfere with daily activities and quality of life. While SjD most frequently affects the glands that produce saliva and tears, more systemic symptoms called extraglandular manifestations are common and may impact multiple organ systems, including joints, lungs, kidneys, and nervous system. Patients with SjD also have a high-risk of developing numerous associated conditions, including up to 20 times higher risk of developing B-cell lymphomas when compared to the general population. Patients with high activity in more than one organ or disease domain have an increased mortality risk of up to five-fold.
