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- Title
Nedosiran: First Approval.
- Authors
Syed, Yahiya Y.
- Abstract
Nedosiran (RIVFLOZA™), a once-monthly subcutaneous small interfering RNA (siRNA) therapy, is being developed by Dicerna Pharmaceuticals, a Novo Nordisk company, for the treatment of primary hyperoxaluria (PH). It reduces oxalate overproduction by inhibiting the expression of the hepatic lactate dehydrogenase (LDH) enzyme. Nedosiran received its first approval on 29 September 2023 in the USA to lower urinary oxalate levels in children aged ≥ 9 years and adults with PH type 1 (PH1) and relatively preserved kidney function [e.g. estimated glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73 m2]. This article summarizes the milestones in the development of nedosiran leading to this first approval for PH1.
- Subjects
DRUG approval; GLOMERULAR filtration rate; SMALL interfering RNA; METABOLIC disorders; LACTATE dehydrogenase; INBORN errors of carbohydrate metabolism; OXALIC acid; DRUG development
- Publication
Drugs, 2023, Vol 83, Issue 18, p1729
- ISSN
0012-6667
- Publication type
Article
- DOI
10.1007/s40265-023-01976-4