Nedosiran in primary hyperoxaluria type 1: a profile of its use

<p><strong>Declarations</strong></p> <p><strong>Funding</strong> The preparation of this review was not supported by any external funding.</p> <p><strong>Authorship and conflict of interest</strong> N. L. France and Y. Y. Syed are salaried employees of Adis International Ltd/Springer Nature and declare no relevant conflicts of interest. All authors contributed to this article and are responsible for its content.</p> <p><strong>Ethics approval, Consent to participate, Consent for publication, Availability of data and material, Code availability</strong> Not applicable.</p> <p>Additional information about this Adis Drug Review can be found <a href="http://www.springer.com/gp/adis/products-services/adis-journals-newsletters/adis-drug-reviews" target="_blank"><strong>here</strong></a>. </p> <p><strong>Abstract</strong></p> <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> <p><strong>©</strong> Springer Nature Switzerland AG 2025</p>