Sibeprenlimab Receives U.S. FDA Breakthrough Therapy Designation for the Treatment of Immunoglobulin A Nephropathy

Otsuka Pharmaceutical Development & Commercialization, Inc., (OPDC); Otsuka Pharmaceutical, Co. Ltd. (Otsuka); and Visterra, Inc., an Otsuka group company announce that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for the investigational drug sibeprenlimab for the treatment of immunoglobulin A nephropathy (IgAN; Berger’s disease).

Sibeprenlimab is a humanized monoclonal antibody that blocks the action of the B-cell growth factor, APRIL (an acronym for a proliferation-inducing ligand), which plays a key role in the development and progression of IgAN. Otsuka and Visterra announced positive results of the Phase 2 ENVISION trial (NCT04287985) for IgAN in November 2023. The trial results were also published in The New England Journal of Medicine. Breakthrough therapy designation is granted by the FDA for a drug intended to treat a serious condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on at least one clinically significant endpoint.  Breakthrough therapy designation allows the agency to prioritize and focus resources on the most promising products for which no satisfactory treatment option exists.

“We are encouraged by the FDA’s decision to grant breakthrough therapy status for the sibeprenlimab program,” said John Kraus, M.D., Ph.D., executive vice president and chief medical officer at Otsuka. “This is an important milestone that recognizes the potential value that this investigational candidate may have in the future for people living with one of the most common causes of kidney failure. Otsuka and Visterra are committed to advancing the Phase 3 trial for sibeprenlimab and we are deeply appreciative to all of the study participants, their caregivers, and the investigators who have contributed so much to this research.”

Immunoglobulin A nephropathy is the most common form of primary glomerulonephritis worldwide and is the most common cause of kidney failure in young adults. The disease is associated with a reduction in life expectancy of 10 years, with at least 30 percent of affected patients progressing to kidney failure within 20 to 30 years, despite optimized standard of care therapy. Current standard of care management is based on renin-angiotensin aldosterone system (RAAS) blockers and adequate blood pressure control, but the risk of kidney failure remains high.

“Sibeprenlimab is the first of several precision therapy biologics designed and engineered by Visterra to treat chronic kidney disease patients with major unmet medical needs,” said Brian J. G. Pereira, M.D., chief executive officer of Visterra Inc. “The results of the Phase 2 trial in patients with IgA nephropathy have been encouraging, and we look forward to completion of the Phase 3 trial and submission of the Biological Licensing Application (BLA) to the US FDA and other global regulatory authorities.”