Nedosiran

Nedosiran is an RNA interference targeting hepatic lactate dehydrogenase, the enzyme responsible for the conversion of glyoxylate to oxalate. Oxalate, particularly calcium oxalate, precipitation is the main cause of kidney stones formation; therefore, blocking the production of oxalate can help alleviate renal symptoms. Nedosiran was approved by the FDA on October 2, 2023, under the brand name RIVFLOZA 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. This approval is based on the favorable results from the pivotal phase 2 PHYOX2 and interim data from the ongoing phase 3 PHYOX3 clinical trials.

Nedosiran is an RNA interference targeting hepatic lactate dehydrogenase, the enzyme responsible for the conversion of glyoxylate to oxalate. Oxalate, particularly calcium oxalate, precipitation is the main cause of kidney stones formation; therefore, blocking the production of oxalate can help alleviate renal symptoms. Nedosiran was approved by the FDA on October 2, 2023, under the brand name RIVFLOZA 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. This approval is based on the favorable results from the pivotal phase 2 PHYOX2 and interim data from the ongoing phase 3 PHYOX3 clinical trials.

RIVFLOZA is indicated 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, e.g., eGFR ≥ 30 mL/min/1.73 m.

• Primary Hyperoxaluria Type 1 (PH1)
• Urine oxalate increased