The approval was supported by the positive findings from the Phase 2 PHYOX 2 clinical trial and interim data from the ongoing Phase 3 PHYOX 3 extension trial
US FDA clears Novo Nordisk's therapy for primary hyperoxaluria type 1. (Credit: julien Tromeur on Unsplash)
Novo Nordisk secured the US Food and Drug Administration (FDA) approval for Rivfloza (nedosiran) injection in children and adults with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function.
Rivfloza injection 80 mg, 128 mg, or 160 mg is a prescription-based once-monthly subcutaneous ribonucleic acid interference (RNAi) therapy.
The FDA has approved the drug to lower urinary oxalate levels in adults and children aged nine years or more with PH1.
The approval was supported by the findings from the Phase 2 PHYOX 2 clinical trial and interim data from the ongoing Phase 3 PHYOX 3 extension trial.
According to the results, PHYOX 2 met its primary endpoint. The patients administered with Rivfloza showed a marked reduction from baseline in 24-hour-urinary oxalate (Uox) excretion from Day 90 to Day 180.
The least-squares (LS) mean between group difference of area under the curve (AUC) was 4976, which was significant between the Rivfloza and placebo groups over the 90 days, the Danish pharmaceutical firm said.
As per the preliminary findings from the PHYOX 3 extension study, 13 patients with PH1 who had received an additional six months of Rivfloza maintained reductions in 24-hour Uox excretion.
Novo Nordisk Strategy and Rare Disease SVP Blandine Lacroix said: “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.
“We look forward to making our first RNAi treatment available to people living with PH1 and the healthcare professionals partnering on their care.”
Rivfloza was created by Dicerna Pharmaceuticals which Novo Nordisk bought in 2021. It further developed its first RNAi treatment using the proprietary GalXC RNAi technology platform.
Novo Nordisk created Rivfloza, the company’s first RNAi treatment, using the proprietary GalXC RNAi technology platform.
The company plans to make Rivfloza accessible to eligible patients by the start of next year.