Novo Nordisk received approval from the U.S. Food and Drug Administration (FDA) on Sept. 29, 2023, for Rivfloza™ (nedosiran) injection. It is indicated for treating patients who are at least nine years old, who have primary hyperoxaluria (PH) type 1 (PH1), and who have relatively preserved kidney function as shown by an estimated glomerular filtration rate (eGFR) of 30mL/min/1.73m2 or greater. A group of rare inherited metabolic conditions, PH results from too much oxalate in the body. Rivfloza uses ribonucleic acid interference (RNAi) to interrupt the formation of oxalate in the body. Recommended once monthly, subcutaneous (SC) dosing for Rivfloza is based on the individual’s weight with patients between nine years and 11 years old who weigh less than 50kg (110 pounds) receiving 3.3mg/kg. Adults and adolescents 12 years and older who weigh less than 50kg get 128mg and all patients who weigh 50kg or more need 160mg. The launch is planned for early 2024, but pricing and distribution information presently are not available. Full prescribing information can be found here.
At a Glance
- Brand (Generic) Name: Rivfloza (nedosiran)
- Manufacturer: Novo Nordisk
- Date Approved: Sept. 29, 2023
- Indication: to lower urinary oxalate levels in patients nine years of age and older who have PH1 with relatively preserved kidney function
- Dosage Forms Available: single-dose vials with 80mg and prefilled single-dose syringes with 128mg or 160mg
- Launch Date: Early in 2024
- Estimated Annual Cost: Not yet known
- The liver produces oxalate, a chemical also contained in some foods, which usually is broken down by enzymes and eliminated mainly through the urine.
- Patients who have PH produce too much oxalate and lack the enzymes needed to metabolize it or both.
- Some patients do not have early symptoms of PH, but recurring kidney and bladder stones — particularly for children, teens, and young adults — often are the first indicator of the condition. Other symptoms can include frequent and/or painful urination, lower back pain, and bloody, muddy-looking, or strong-smelling urine.
- Oxalate accumulation damages the kidneys, eventually leading to end-stage renal disease (ESRD) and kidney failure. Other problems, such as anemia, delayed growth, eye problems, and heart disease can develop as oxalate crystals deposit in various parts of the body.
- Novo Nordisk estimates that as many as 2,000 patients in the U.S. have PH1.
- Rivfloza interferes with the messenger RNA that encodes for a protein critical to the formation of oxalate to reduce the amounts generated in the liver.
- Clinical trials of Rivfloza included children as young as six years old. In the randomized, double-blinded, placebo-controlled PHYOX2 Study, excretion of oxalate reached normal or near normal levels for 64.7% of patients using Rivfloza to treat PH1 after three months of treatment, as compared to none of the patients in the placebo group.
- Commonly experienced side effects included bruises, pain, rash, redness, or other reactions at the site of the injection.
- Oxlumo™ (lumasiran – Alnylam Pharmaceuticals) injection was FDA-approved in 2020 to treat PH1. Its dosing also depends on the patient’s weight with SC injections administered once a month for three doses and then switched to once every three months.
- Other treatment for PH1 centers on avoiding oxalate accumulation with calcium, pyridoxine, and potassium citrate supplementation, staying adequately hydrated, and restricting intake of vitamins C and D. Some patients may need dialysis to filter out excessive oxalate, and some may need kidney/liver transplants. Rivfloza was approved under the FDA’s Priority Review system.