FDA Accepts, Grants Priority Review to Sibeprenlimab BLA for IgA Nephropathy

The US Food and Drug Administration (FDA) has accepted and granted priority review to Otsuka Pharmaceutical Development & Commercialization’s Biologics License Application (BLA) for sibeprenlimab for the treatment of IgA nephropathy (IgAN).1

Announced on May 27, 2025, the BLA acceptance is supported by data from the phase 3 VISIONARY trial, which met its primary endpoint at the prespecified interim analysis, as well as results from the Phase 2 ENVISION clinical trial. The BLA has been granted priority review and a Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2025.1

“If approved, sibeprenlimab would enable individuals living with IgA nephropathy to self-inject once every 4 weeks,” John Kraus, MD, PhD, executive vice president and chief medical officer of Otsuka, said in a press release.1 “We are thankful to share a potential treatment that could offer important clinical benefits and convenience to those living with this disease.”

An investigational monoclonal antibody that selectively inhibits the activity of A PRoliferation-Inducing Ligand (APRIL), sibeprenlimab, formerly known as VIS649, was designed and engineered by Visterra, a wholly owned subsidiary of Otsuka, which also conducted pre-clinical and early-stage trials of sibeprenlimab.1,2

By binding and inhibiting APRIL, sibeprenlimab may help reduce IgA and Gd-IgA1 levels while addressing one of the IgAN-specific drivers for nephron loss. By reducing the production of Gd-IgA1, sibeprenlimab may help slow kidney damage and progression toward end-stage kidney disease.1,2

Sibeprenlimab is a single-dose prefilled syringe for subcutaneous injection every 4 weeks intended for self-administration and was previously granted Breakthrough Therapy designation for the treatment of IgAN in 2024 based on favorable results from the phase 2 ENVISION trial.1,2

Now, sibeprenlimab is being evaluated in the multicenter, randomized, double-blind, placebo-controlled VISIONARY trial, which enrolled approximately 530 adult patients with IgAN who were receiving standard-of-care therapy, defined as maximally tolerated ACE inhibitor or ARB +/- SGLT2 inhibitor.2

The primary efficacy endpoint is the change in 24-hour uPCR at 9 months compared with baseline. The secondary endpoint is the annualized slope of eGFR estimated over ~24 months. At the prespecified interim analysis, sibeprenlimab demonstrated a statistically significant and clinically meaningful reduction in 24-hour uPCR after 9 months of treatment.2

As described in the press release from Otsuka, the BLA has been granted priority review and assigned a PDUFA target action date of November 28, 2025.1

References

1. Businesswire. Otsuka Announces FDA Acceptance and Priority Review of Biologics License Application (BLA) for Sibeprenlimab in the Treatment of Immunoglobulin A Nephropathy (IgAN). May 27, 2025. Accessed May 27, 2025. https://www.businesswire.com/news/home/20250527959047/en/Otsuka-Announces-FDA-Acceptance-and-Priority-Review-of-Biologics-License-Application-BLA-for-Sibeprenlimab-in-the-Treatment-of-Immunoglobulin-A-Nephropathy-IgAN

2. Brooks A. Otsuka Files BLA for Sibeprenlimab for IgA Nephropathy. HCPLive. March 31, 2025. Accessed May 27, 2025. https://www.hcplive.com/view/otsuka-files-bla-sibeprenlimab-iga-nephropathy