FDA approves Rivfloza for primary hyperoxaluria type 1
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Key takeaways:
- During clinical trials, patients treated with Rivfloza had a significant reduction in 24-hour urinary oxalate excretion.
- Novo Nordisk plans to make the drug available in early 2024.
The FDA approved Rivfloza to treat primary hyperoxaluria type 1 in patients aged 9 years and older with relatively preserved kidney function, according to a press release.
“The patients who will benefit from these drugs are those with [the] genetically mediated disease,” David S. Goldfarb, MD, clinical chief in the nephrology division at the NYU Langone Medical Center, and professor of medicine and physiology at the university’s Grossman School of Medicine, told Healio. “Most stone-formers make calcium oxalate stones, with some contribution of oxalate from dietary intake.”
Nedosiran (Rivfloza, Novo Nordisk) is an injection available in 80 mg, 128 mg or 160 mg doses, administered subcutaneously once a month. It operates as a ribonucleic acid interference (RNAi) therapy, designed to lower urinary oxalate levels, the researchers noted.
“People with [primary hyperoxaluria (PH)] have an abnormality in one of three proteins in the liver that leads to excess oxalate synthesis,” Goldfarb said. “The kidney is an innocent bystander, with result of kidney stones and kidney failure. Rivfloza is indicated for patients with PH1.”
The approval comes after results of a phase 2 clinical trial, PHYOX2, and interim findings from the ongoing phase 3 extension study, PHYOX3. In the PHYOX2 trial, patients treated with Rivfloza experienced a significant reduction in 24-hour urinary oxalate excretion from day 90 to 180 mg compared with a placebo group. The most common adverse reaction reported was injection site reactions, which occurred in more than 20% of patients. Interim results from the PHYOX3 extension indicate that the reductions in 24-hour urinary oxalate excretion were consistently maintained in 13 patients with PH1 who received an additional 6 months of treatment with Rivfloza.
“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,” Blandine Lacroix, senior vice president of strategy and rare disease at the company, said in a press release. “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 health care professionals partnering on their care.”
On whether the drug will restore oxalate excretion to standard levels, Goldfarb said a “significant proportion of patients do have normalization of the 24-hour oxalate excretion.” However, it may not be “absolutely necessary to have a ‘normal’ value. Any reduction is meaningful and likely to be associated with less progressive CKD and fewer stones.”
The long-term issue, he added, “will be whether lowering oxalate excretion will reduce the progression of CKD. Ultimately, avoiding the need for liver and kidney transplant would be the desirable goal.”
Rivfloza was developed by Dicerna Pharmaceuticals, which was acquired by Nordisk in 2021. Nordisk plans to make the drug available in early 2024, according to the release.
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