FDA Approves Rivfloza™ (nedosiran) for the Treatment of Primary Hyperoxaluria Type 1 (PH1)
Novo Nordisk announced the U.S. Food and Drug Administration has approved RivflozaTM (nedosiran) injection 80 mg, 128 mg, or 160 mg, a once-monthly subcutaneous ribonucleic acid interference (RNAi) therapy, to lower urinary oxalate levels in children 9 years of age and older and adults with primary hyperoxaluria type 1 (PH1) 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% 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 over 2,000 people are living with PH1.
RivflozaTM, the first RNAi therapeutic by Novo Nordisk, was developed using the proprietary GalXCTM RNAi technology platform. RivflozaTM 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. RivflozaTM was developed by Dicerna Pharmaceuticals, Inc. which was acquired by Novo Nordisk in 2021.
Novo Nordisk plans to make RivflozaTM available for eligible patients in early 2024. The most common side effects of RivflozaTM include injection site reactions, such as reddening, pain, bruising, rash, or dimple at the site of injection.
