<p>Nedosiran (Rivfloza^®), a lactate dehydrogenase A-directed small interfering RNA (siRNA), is a valuable addition to the treatment options available for patients with primary hyperoxaluria type 1 (PH1). It is the second siRNA therapy approved for use in PH1 (following lumasiran) in the USA. Nedosiran is administered once monthly via subcutaneous injection by a healthcare professional, caregiver or patient aged ≥ 12 years. Results from PHYOX2, a 6-month, multinational, randomized, double-blind, placebo-controlled, pivotal phase 2 trial, show that nedosiran produces significantly greater reductions in urinary oxalate excretion than placebo in patients with PH1, with approximately two-thirds of patients achieving normal or near-normal levels of urinary oxalate excretion. In the ongoing phase 3 PHYOX3 trial, the efficacy of nedosiran was sustained for ≥ 30 months. Nedosiran is generally well tolerated, with adverse events typically being mild to moderate in severity. The most common treatment-related adverse events are injection site reactions.</p>