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Synonyms: DCR-PHXC | NN-7022 | Rivfloza®
nedosiran is an approved drug (FDA (2023))
Compound class:
Nucleic acid
Comment: Nedosiran (DCR-PHXC) is a synthetic double-stranded siRNA drug. It contains a target-selective guide strand, plus a passenger strand that is attached to an extended nucleotide region that acts as a dicer substrate siRNA which is conjugated with four N-acetylgalactosamine (GalNAc) sugars. These modifications act to both stabilise the siRNA and optimise orientation for presentation to hepatocytes [2,4]. Nedosiran targets lactate dehydrogenase A (LDHA) mRNA as a mecahnism to reduce urinary oxalate levels as a treatment for primary hyperoxaluria.
We have been unable to locate a full chemical SMILES for the drug, or its HELM notation. |
| No information available. |
Summary of Clinical Use  |
| Nedosiran (DCR-PHXC) was progressed to clinical evaluation for efficacy in patients with primary hyperoxaluria (PH). It was approved by the FDA in September 2023 to treat PH type 1 [3]. |
| Clinical Trials | |||||
| Clinical Trial ID | Title | Type | Source | Comment | References |
| NCT04580420 | Safety & Efficacy of DCR-PHXC in Patients With PH1 and ESRD | Phase 2 Interventional | Novo Nordisk A/S | ||
| NCT05001269 | Nedosiran in Pediatric Patients From Birth to 11 Years of Age With PH and Relatively Intact Renal Function | Phase 2 Interventional | Novo Nordisk A/S | ||
| NCT03847909 | A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 | Phase 2 Interventional | Novo Nordisk A/S | 1 | |
| NCT04042402 | Long Term Extension Study in Patients With Primary Hyperoxaluria | Phase 3 Interventional | Novo Nordisk A/S | ||
| External links |
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For extended ADME data see the following: Drugs.com |
