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First of Its Kind Treatment for Primary Hyperoxaluria Wins Approval, Holds Promise

  • 1 Ray Bignall II, MD, is Director, Kidney Health Advocacy and Community Engagement, Division of Nephrology and Hypertension, Nationwide Children's Hospital, and Clinical Assistant Professor of Pediatrics, The Ohio State University College of Medicine, Columbus, OH.
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In November 2020, the US Food and Drug Administration (FDA) approved lumasiran (brand name “Oxlumo”), the first medical therapy specifically for the management of primary hyperoxaluria type 1 (PH1), a rare and life-threatening disease that often progresses to kidney failure. This announcement may represent a breakthrough, not only in the treatment of PH1 but also in drug development for a host of rare kidney diseases.

PH1 is caused by a congenital defect in the hepatic enzyme alanine glyoxylate aminotransferase, resulting in a failure to metabolize glyoxylate to glycine and the toxic accumulation of oxalate. The buildup of unmetabolized oxalate leads