FDA Grants Priority Review to Otsuka’s Sibeprenlimab for IgAN

Otsuka Pharmaceutical Development & Commercialization, Inc. and Otsuka Pharmaceutical Co., Ltd. (Otsuka) announce the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) for sibeprenlimab, an investigational monoclonal antibody that selectively inhibits the activity of APRIL (A PRoliferation-Inducing Ligand) in adults with immunoglobulin A nephropathy (IgAN). APRIL plays a key role in the pathogenesis of IgAN as explained by the 4-hit process, in which pathogenic galactose-deficient IgA (Gd-IgA1) is produced, leading to the synthesis of autoantibodies against Gd-IgA1, immune complex formation, and deposition in the glomerular mesangium. Sibeprenlimab is a single-dose prefilled syringe for subcutaneous injection every four weeks intended for self-administration, providing patients the convenience of at-home delivery.

The BLA is supported by the Phase 3 VISIONARY clinical trial (NCT05248646), which met its primary endpoint at the prespecified interim analysis, and results from the Phase 2 ENVISION clinical trial (NCT04287985). Sibeprenlimab demonstrated a statistically significant and clinically meaningful reduction in 24-hour uPCR after nine months of treatment compared to placebo in the Phase 3 VISIONARY trial.

The BLA has been granted priority review and a Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2025.

“Over the past decade, Otsuka has consistently approached difficult-to-treat diseases in nephrology with scientific and clinical innovation, seeking to provide crucial advancements for underserved patients with complex conditions like IgA nephropathy,” said John Kraus, M.D., Ph.D., executive vice president and chief medical officer, Otsuka Pharmaceutical Development & Commercialization, Inc. “If approved, sibeprenlimab would enable individuals living with IgA nephropathy to self-inject once every 4 weeks. We are thankful to share a potential treatment that could offer important clinical benefits and convenience to those living with this disease.”

Sibeprenlimab has Breakthrough Therapy designation for the treatment of IgAN based on the favorable results from the Phase 2 (ENVISION) trial (NCT04287985).

IgAN is a progressive, autoimmune, chronic kidney disease that can lead to end-stage kidney disease (ESKD) over the lifetime of most patients under current optimized standard care.

Targeting APRIL represents a potential therapeutic strategy to disrupt IgAN’s pathogenesis via the 4-hit process, which may reduce production of immune complexes, which can deposit into kidney tissue, and may lead to ESKD and the need for transplantation.