Nedosiran, sold under the brand name Rivfloza, is a medication used for the treatment of primary hyperoxaluria.[1] It is an LDHA-directed small interfering RNA developed by Dicerna Pharmaceuticals.[1][2]
| Clinical data | |
|---|---|
| Trade names | Rivfloza |
| Other names | DCR-PHXC |
| AHFS/Drugs.com | Monograph |
| License data | |
| Routes of administration | Subcutaneous |
| ATC code |
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| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
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 US Food and Drug Administration (FDA) considers it to be a first-in-class medication.[6]
Medical uses
editPrimary hyperoxaluria type 1 is a rare disease in which urine oxalate is too high, which over time can harm the kidneys.[3]
Nedosiran is indicated to lower urinary oxalate levels in people nine years of age and older with primary hyperoxaluria type 1 and relatively preserved kidney function.[1][3]
History
editThe 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]
Society and culture
editLegal status
editThe FDA granted the application for nedosiran breakthrough therapy and orphan drug designations.[6] Nedosiran was approved for medical use in the United States in September 2023.[1][3]
Names
editNedosiran is the international nonproprietary name.[7]
References
edit- ^ a b c d e f "Rivfloza (nedosiran) injection, for subcutaneous use" (PDF). Archived (PDF) from the original on 1 October 2023. Retrieved 1 October 2023.
- ^ "Nedosiran". Dicerna Pharmaceuticals. 17 August 2021. Archived from the original on 26 July 2023. Retrieved 1 October 2023.
- ^ a b c d e f g h i j "Drug Trials Snapshots: Rivfloza". U.S. Food and Drug Administration (FDA). 29 September 2023. Retrieved 15 July 2024.
This article incorporates text from this source, which is in the public domain.
- ^ "Rivfloza: FDA-Approved Drugs". U.S. Food and Drug Administration (FDA). Archived from the original on 2 October 2023. Retrieved 1 October 2023.
- ^ "Drug Approval Package: Rivfloza". U.S. Food and Drug Administration (FDA). 18 October 2023. Archived from the original on 10 January 2024. Retrieved 10 January 2024.
- ^ a b New Drug Therapy Approvals 2023 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2024. Archived from the original on 10 January 2024. Retrieved 9 January 2024.
- ^ World Health Organization (2021). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 85". WHO Drug Information. 35 (1). hdl:10665/340684.
Further reading
edit- Forbes TA, Brown BD, Lai C (June 2022). "Therapeutic RNA interference: A novel approach to the treatment of primary hyperoxaluria". British Journal of Clinical Pharmacology. 88 (6): 2525–2538. doi:10.1111/bcp.14925. PMC 9291495. PMID 34022071.
- Liu A, Zhao J, Shah M, Migliorati JM, Tawfik SM, Bahal R, et al. (November 2022). "Nedosiran, a Candidate siRNA Drug for the Treatment of Primary Hyperoxaluria: Design, Development, and Clinical Studies". ACS Pharmacology & Translational Science. 5 (11): 1007–1016. doi:10.1021/acsptsci.2c00110. PMC 9667536. PMID 36407951.
External links
edit- Clinical trial number NCT03847909 for "A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 (PHYOX2)" at ClinicalTrials.gov
- Clinical trial number NCT04042402 for "Long Term Extension Study in Patients With Primary Hyperoxaluria (PHYOX3)" at ClinicalTrials.gov