Nedosiran is an antisense oligonucleotide used to treat primary hyperoxaluria type 1 in adults and high oxalate levels in children. Nedosiran is an RNA interference targeting hepatic lactate dehydrogenase, the enzyme responsible for the conversion of glyoxylate to oxalate.
- Mechanism
- Curator reviewed · 2 references
Nedosiran is a double-stranded siRNA, conjugated to GalNAc aminosugar residues. After subcutaneous administration, the GalNAc-conjugated sugars bind to asialoglycoprotein receptors (ASGPR) to deliver nedosiran to hepatocytes.
Nedosiran reduces levels of hepatic lactate dehydrogenase (LDH) via the degradation of LDHA messenger ribonucleic acid (mRNA) in hepatocytes through RNA interference. The reduction of hepatic LDH by nedosiran reduces the production of oxalate by the liver, thereby reducing subsequent oxalate burden.
- Primary indication
- 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 m2.Curator reviewed · 2 structured indications
- First approval
- United States, 2024
- Also known as
- DCR-PHXC free acid
- Code names
- DCR-PHXC
- Brand names
Resolves to
What you can answer from here — as of September 12, 2026
Which drugs share a target with Nedosiran, and which of those have an active Phase 3 trial?
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