Sibeprenlimab Granted Breakthrough Therapy Designation by U.S. FDA for Immunoglobulin A Nephropathy
Saturday, February 17, 2024
Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC), alongside Otsuka Pharmaceutical Co. Ltd. (Otsuka) and Visterra, Inc., a subsidiary of Otsuka, have announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for sibeprenlimab, an investigational drug aimed at treating immunoglobulin A nephropathy (IgAN), also known as Berger’s disease.
Sibeprenlimab, a humanized monoclonal antibody, works by inhibiting the activity of the B-cell growth factor APRIL (a proliferation-inducing ligand), which is pivotal in the development and progression of IgAN. The Phase 2 ENVISION trial (NCT04287985) for IgAN, conducted by Otsuka and Visterra, yielded positive results in November 2023, with findings also published in The New England Journal of Medicine. This designation by the FDA recognizes the potential significance of sibeprenlimab in offering substantial improvement over existing therapies for a serious condition like IgAN.
Otsuka, expressed encouragement at the FDA's decision, emphasizing the potential impact of sibeprenlimab on individuals grappling with one of the most prevalent causes of kidney failure. He reiterated Otsuka and Visterra's commitment to advancing the Phase 3 trial for sibeprenlimab, expressing gratitude to study participants, caregivers, and investigators for their invaluable contributions.
IgAN stands as the primary form of glomerulonephritis worldwide and is a leading cause of kidney failure among young adults. Despite optimized standard care therapy, a significant proportion of patients experience disease progression, highlighting the urgent need for effective treatments. Sibeprenlimab, as the first in a line of precision therapy biologics developed by Visterra, holds promise in addressing the unmet medical needs of chronic kidney disease patients.
Sibeprenlimab, previously known as VIS649, is an investigational humanized IgG2 monoclonal antibody designed to reduce the production of Gd-IgA1 by targeting APRIL, a key signaling molecule implicated in IgA and Gd-IgA1 production. By neutralizing APRIL, sibeprenlimab aims to lower IgA and Gd-IgA1 levels, potentially leading to reduced auto-antibody production, decreased immune complex deposits in the kidneys, and mitigated kidney inflammation. Ultimately, sibeprenlimab aims to prevent further kidney damage and halt the progression to end-stage kidney disease.
The cytokine APRIL, belonging to the tumor necrosis factor (TNF) superfamily, is recognized for its role in B-cell signaling, particularly in IgA class switch recombination and the production and survival of IgA-secreting plasma cells. Its involvement in IgAN pathogenesis underscores its significance as a therapeutic target in addressing this condition.
Source: businesswire.com
