U of M Medical School research team finds novel drug improves outcomes for patients with rare kidney disorder

MINNEAPOLIS/ST. PAUL (11/03/2023) — Focal segmental glomerulosclerosis (FSGS) is a rare kidney disorder that affects children and adults, and can lead to kidney failure. New findings from a team led by the University of Minnesota Medical School show patients with FSGS who were treated with the medication sparsentan experienced improved kidney function—making it a potential new treatment option for the disorder.

The research, published today in the New England Journal of Medicine, suggests sparsentan may provide kidney protection by significantly reducing excess protein in urine — known as proteinuria, a proven indicator of kidney damage. 

“FSGS is a frustrating disease for children to deal with and affects their quality of life,” said Michelle Rheault, MD, professor at the U of M Medical School and pediatric nephrologist with M Health Fairview. “We're committed to offering our patients with kidney disease access to new treatments through our clinical trial options.”

Clinically, reduction of proteinuria and delaying time to kidney failure are critically important for patients. FSGS patients in this study who were treated with sparsentan through two years of treatment had lower protein in their urine and were more likely to achieve complete remission compared to patients treated with irbesartan, the current standard treatment.

Although the endpoint for glomerular filtration rate—which measures how well kidneys filter blood—was not achieved after two years of treatment, other improvements were seen, including: 

• Significant proteinuria reduction
• Higher rates of partial or complete remission
• Lower rates of reaching end-stage kidney disease

These findings suggest sparsentan could be a potential new treatment option for FSGS. Clinically, reduction of proteinuria and delaying time to kidney failure are critically important for patients. 

Further analysis is ongoing to determine which patients with FSGS may benefit the most from sparsentan.

This research was funded by Travere Therapeutics—the maker of sparsentan.

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About the University of Minnesota Medical School
The University of Minnesota Medical School is at the forefront of learning and discovery, transforming medical care and educating the next generation of physicians. Our graduates and faculty produce high-impact biomedical research and advance the practice of medicine. We acknowledge that the U of M Medical School, both the Twin Cities campus and Duluth campus, is located on traditional, ancestral and contemporary lands of the Dakota and the Ojibwe, and scores of other Indigenous people, and we affirm our commitment to tribal communities and their sovereignty as we seek to improve and strengthen our relations with tribal nations. For more information about the U of M Medical School, please visit med.umn.edu.