Nipocalimab gets FDA breakthrough therapy nod as first for indication.
Nipocalimab has been granted breakthrough therapy designation by the US Food and Drug Administration for the treatment of adults living with moderate-to-severe Sjögren’s disease.
The debilitating and chronic autoantibody disease has no approved advanced treatments available, and this is the first time an investigational therapy has secured BTD for Sjögren’s (SjD).
However, it is the second BTD granted by the FDA for nipocalimab – the first was in February this year for the treatment of alloimmunised pregnant individuals at high risk of severe haemolytic disease of the foetus and newborn.
Terence Rooney vice president and Rheumatology, Immunology Disease Area Leader, at Johnson & Johnson Innovative Medicine said the announcement marks an important step forward in the continued research and development of nipocalimab.
He said it was 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 Sjögren’s disease,” he said.
“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.”
Further clinical trials and regulatory reviews are expected to follow that will determine the future availability of nipocalimab for SjD patients.
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 features 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 results were also published in the Annals of the Rheumatic Diseases.
“DAHLIAS is the first study of a FcRn blocker in SjD and shows that nipocalimab treatment led to significant improvement over placebo in clinESSDAI and similar trends in other key efficacy endpoints at Wk 24 and is well-tolerated,” the authors concluded.
“These results demonstrate the mechanistic relevance of FcRn inhibition in SjD, indicating the potential pathogenicity of IgG AAb. Together, these findings establish proof of concept for nipocalimab in SjD and support further evaluation in patients with moderate-to-severe AAb-positive 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, and 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.