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

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posted on 2025-01-14, 20:35 authored by Nicole L. France, Yahiya Y. Syed

Declarations

Funding The preparation of this review was not supported by any external funding.

Authorship and conflict of interest 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.

Ethics approval, Consent to participate, Consent for publication, Availability of data and material, Code availability Not applicable.

Additional information about this Adis Drug Review can be found here

Abstract

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.

© Springer Nature Switzerland AG 2025

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