| Clinical data | |
|---|---|
| Trade names | Rivfloza |
| Other names | DCR-PHXC |
| AHFS/Drugs.com | Monograph |
| License data | |
| Routes of administration | Subcutaneous |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
Nedosiran, sold under the brand nameRivfloza, is amedication used for the treatment ofprimary hyperoxaluria.[1] It is anLDHA-directedsmall interfering RNA developed by Dicerna Pharmaceuticals.[1][2]
The most common side effects include injection site reactions.[3]
Nedosiran was approved for medical use in the United States in September 2023.[1][4][5][3] The USFood and Drug Administration (FDA) considers it to be afirst-in-class medication.[6]
Primary hyperoxaluria type 1 is a rare disease in which urine oxalate is too high, which over time can harm the kidneys.[3]
Nedosiran isindicated to lower urinaryoxalate levels in people nine years of age and older with primary hyperoxaluria type 1 and relatively preserved kidney function.[1][3]
The US FDA approved nedosiran based on evidence from a clinical trial which included 29 participants with primary hyperoxaluria type 1.[3] Nedosiran was evaluated in one clinical trial of 29 participants with primary hyperoxaluria type 1 who were nine years of age and older.[3] Participants randomly received either nedosiran or placebo injections once a month for six months.[3] Neither the participants nor the healthcare providers knew which treatment was being given.[3] The benefit of nedosiran was assessed by measuring the amount of oxalate in the urine and comparing it to placebo. The trial was conducted at 19 sites in 11 countries in North America, Europe, Asia, and Australia.[3]
The FDA granted the application for nedosiranbreakthrough therapy andorphan drug designations.[6] Nedosiran was approved for medical use in the United States in September 2023.[1][3]
Nedosiran is theinternational nonproprietary name.[7]
This article incorporates text from this source, which is in thepublic domain.