View source for Nedosiran

{{Short description|Medication}}
{{Use American English|date=October 2023}}
{{Use dmy dates|date=October 2023}}
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| pronounce         = 
| tradename         = Rivfloza 
| Drugs.com         = {{drugs.com|monograph|nedosiran}}
| MedlinePlus       = 
| DailyMedID        = Nedosiran
| pregnancy_AU      = <!-- A / B1 / B2 / B3 / C / D / X -->
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| routes_of_administration = [[Subcutaneous administration|Subcutaneous]]
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| ATC_prefix        = None <!-- Scheduled to be A16AX25 in 2025 -->
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| legal_AU          = <!-- S2, S3, S4, S5, S6, S7, S8, S9 or Unscheduled -->
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| legal_UK          = <!-- GSL, P, POM, CD, CD Lic, CD POM, CD No Reg POM, CD (Benz) POM, CD (Anab) POM or CD Inv POM / Class A, B, C -->
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| legal_US          = Rx-only
| legal_US_comment  = <ref name="Rivfloza FDA label">{{Cite web |url=https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf |title=Rivfloza (nedosiran) injection, for subcutaneous use |access-date=1 October 2023 |archive-date=1 October 2023 |archive-url=https://web.archive.org/web/20231001235147/https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf |url-status=live }}</ref>
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| CAS_number        = 2266591-83-5
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| DrugBank          = DB17635
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| UNII              = 13U9R5J3WL
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| synonyms          = DCR-PHXC

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'''Nedosiran''', sold under the brand name '''Rivfloza''', is a [[medication]] used for the treatment of [[primary hyperoxaluria]].<ref name="Rivfloza FDA label" /> It is an [[Lactate dehydrogenase A|LDHA]]-directed [[small interfering RNA]] developed by Dicerna Pharmaceuticals.<ref name="Rivfloza FDA label" /><ref>{{cite web | title=Nedosiran | website=Dicerna Pharmaceuticals | date=17 August 2021 | url=https://dicerna.com/pipeline/nedosiran/ | access-date=1 October 2023 | archive-date=26 July 2023 | archive-url=https://web.archive.org/web/20230726061616/https://dicerna.com/pipeline/nedosiran/ | url-status=live }}</ref>

The most common side effects include injection site reactions.<ref name="Rivfloza FDA snapshot" />

Nedosiran was approved for medical use in the United States in September 2023.<ref name="Rivfloza FDA label" /><ref>{{cite web | title=Rivfloza: FDA-Approved Drugs | website=U.S. [[Food and Drug Administration]] (FDA) | url=https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=215842 | access-date=1 October 2023 | archive-date=2 October 2023 | archive-url=https://web.archive.org/web/20231002035105/https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=215842 | url-status=live }}</ref><ref name="Drug Approval Package: Rivfloza">{{cite web | title=Drug Approval Package: Rivfloza | website=U.S. [[Food and Drug Administration]] (FDA) | date=18 October 2023 | url=https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/215842Orig1s000TOC.cfm | access-date=10 January 2024 | archive-date=10 January 2024 | archive-url=https://web.archive.org/web/20240110053505/https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/215842Orig1s000TOC.cfm | url-status=live }}</ref><ref name="Rivfloza FDA snapshot">{{cite web | title=Drug Trials Snapshots: Rivfloza | website=U.S. [[Food and Drug Administration]] (FDA) | date=29 September 2023 | url=https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-rivfloza | access-date=15 July 2024}} {{PD-notice}}</ref> The US [[Food and Drug Administration]] (FDA) considers it to be a [[first-in-class medication]].<ref name="New Drug Therapy Approvals 2023">{{cite report | title=New Drug Therapy Approvals 2023 | website=U.S. [[Food and Drug Administration]] (FDA) | date=January 2024 | url=https://www.fda.gov/media/175253/download | format=PDF | access-date=9 January 2024 | archive-url=https://web.archive.org/web/20240110032419/https://www.fda.gov/media/175253/download | archive-date=10 January 2024 | url-status=live }}</ref>

== Medical uses ==
Primary hyperoxaluria type 1 is a rare disease in which urine oxalate is too high, which over time can harm the kidneys.<ref name="Rivfloza FDA snapshot" />

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.<ref name="Rivfloza FDA label" /><ref name="Rivfloza FDA snapshot" />

== History ==
The US FDA approved nedosiran based on evidence from a clinical trial which included 29 participants with primary hyperoxaluria type 1.<ref name="Rivfloza FDA snapshot" /> Nedosiran was evaluated in one clinical trial of 29 participants with primary hyperoxaluria type 1 who were nine years of age and older.<ref name="Rivfloza FDA snapshot" /> Participants randomly received either nedosiran or placebo injections once a month for six months.<ref name="Rivfloza FDA snapshot" /> Neither the participants nor the healthcare providers knew which treatment was being given.<ref name="Rivfloza FDA snapshot" /> 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.<ref name="Rivfloza FDA snapshot" />

== Society and culture ==
=== Legal status ===
The FDA granted the application for nedosiran [[breakthrough therapy]] and [[orphan drug]] designations.<ref name="New Drug Therapy Approvals 2023" /> Nedosiran was approved for medical use in the United States in September 2023.<ref name="Rivfloza FDA label" /><ref name="Rivfloza FDA snapshot" />

=== Names ===
Nedosiran is the [[international nonproprietary name]].<ref>{{cite journal | vauthors = ((World Health Organization)) | year = 2021 | title = International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 85 | journal = WHO Drug Information | volume = 35 | issue = 1 | hdl = 10665/340684 | hdl-access = free | author-link = World Health Organization }}</ref>

== References ==
{{reflist}}

== Further reading ==
{{refbegin}}
* {{cite journal | vauthors = Forbes TA, Brown BD, Lai C | title = Therapeutic RNA interference: A novel approach to the treatment of primary hyperoxaluria | journal = British Journal of Clinical Pharmacology | volume = 88 | issue = 6 | pages = 2525–2538 | date = June 2022 | pmid = 34022071 | pmc = 9291495 | doi = 10.1111/bcp.14925 }}
* {{cite journal | vauthors = Liu A, Zhao J, Shah M, Migliorati JM, Tawfik SM, Bahal R, Rasmussen TP, Manautou JE, Zhong XB | title = Nedosiran, a Candidate siRNA Drug for the Treatment of Primary Hyperoxaluria: Design, Development, and Clinical Studies | journal = ACS Pharmacology & Translational Science | volume = 5 | issue = 11 | pages = 1007–1016 | date = November 2022 | pmid = 36407951 | pmc = 9667536 | doi = 10.1021/acsptsci.2c00110 }}
{{refend}}

== External links ==
* {{ClinicalTrialsGov|NCT03847909|A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 (PHYOX2)}}
* {{ClinicalTrialsGov|NCT04042402|Long Term Extension Study in Patients With Primary Hyperoxaluria (PHYOX3)}}

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[[Category:Small interfering RNA]]
[[Category:Orphan drugs]]