Novo Nordisk has received approval from the US Food and Drug Administration (FDA) for Rivfloza (nedosiran) by injection to reduce urinary oxalate levels in children aged nine years and above, and adults with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function. PH is a rare genetic disease, which causes oxalate overproduction by the liver and affects one in 38,600 individuals globally.
The approval is indicated for Rivfloza 80mg, 128mg or 160mg, a monthly subcutaneous ribonucleic acid interference therapy.
It was based on data from the pivotal Phase II PHYOX2 clinical study and interim results from the ongoing Phase III PHYOX3 extension study.
Developed using the GalXCRNAi technology platform, Rivfloza inhibits the expression of the liver enzyme, lactate dehydrogenase.
The PHYOX 2 trial, which achieved its primary endpoint, demonstrated that patients who received Rivfloza attained a marked reduction from baseline in 24-hour urinary oxalate excretion from day 90 to day 180.
Novo Nordisk strategy and rare disease senior vice-president Blandine Lacroix stated: “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.
“We are committed to driving change on behalf of people living with rare diseases and helping address the significant unmet needs of the PH1 community.”
Dicerna Pharmaceuticals, which was acquired in 2021 by Novo Nordisk, developed Rivfloza. Novo Nordisk aims to provide the therapy for eligible patients in early 2024.
