FDA Approves Novo Nordisk’s RNAi Therapy for Rare Kidney Disease
October 3, 2023
Rare Daily Staff
The U.S. Food and Drug Administration approved Novo Nordisk’s Rivfloza to lower urinary oxalate levels in children 9 years of age and older and adults with the rare kidney disease primary hyperoxaluria type 1 and relatively preserved kidney function.
Primary hyperoxaluria (PH) is a rare genetic disease that causes overproduction of oxalate by the liver that is estimated to affect 1 in 38,600 individuals worldwide. PH1 is the most clinically prevalent (roughly 80 percent of PH patients) and severe of the three subtypes of PH. PH1 is a progressive metabolic disorder that primarily affects the kidneys and can lead to progressive kidney damage. In the U.S., it is estimated that more than 2,000 people are living with PH1. Rivfloza (nedosiran) is a once-monthly subcutaneous ribonucleic acid interference (RNAi) therapy.
“The FDA approval of Rivfloza builds on Novo Nordisk’s legacy of advancing research, fostering innovation and creating strategic partnerships to expand treatment options in rare diseases,” said Blandine Lacroix, senior vice president, Strategy and Rare Disease at Novo Nordisk.
The approval is based on the results of the pivotal phase 2 PHYOXTM2 clinical trial and interim data from the ongoing phase 3 PHYOXTM3 extension study. PHYOXTM2 met its primary endpoint, showing that patients treated with Rivfloza achieved a marked reduction from baseline in 24 hour-urinary oxalate (Uox) excretion from Day 90 to Day 180. The percent change from baseline in 24-hour Uox was measured using an area under the curve (AUC) analysis. The least-squares (LS) mean between group difference of AUC24-hour Uox was 4976, which was significant between the Rivfloza and placebo groups over the 90 days. The most common adverse reaction (reported in ≥20 percent of patients) is injection site reactions. Interim results from the PHYOXTM3 extension study showed reductions in 24-hour Uox excretion were maintained in the 13 patients with PH1 who had received an additional six months of treatment with Rivfloza.
Rivfloza, the first RNAi therapeutic by Novo Nordisk, was acquired by the company when it bought Dicerna Pharmaceuticals in 2021. It is designed to inhibit the expression of liver enzyme lactate dehydrogenase, a liver enzyme that catalyzes the final common step in the glyoxylate metabolism pathway which leads to the oxalate overproduction in patients with PH1.
Photo: Blandine Lacroix, senior vice president, Strategy and Rare Disease at Novo Nordisk.
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