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Nedosiran
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<h2 id="medical-uses">Medical uses</h2> <p>Primary hyperoxaluria type 1 is a rare disease in which urine oxalate is too high, which over time can harm the kidneys.<a class="footnote-ref" id="fnref:10" href="#fn:10"><sup>10</sup></a> </p><p>Nedosiran is <a href="/facts/Indicated/DLdGwxDY">indicated</a> to lower urinary <a href="/facts/Oxalate/u0Gdem0A">oxalate</a> levels in people nine years of age and older with primary hyperoxaluria type 1 and relatively preserved kidney function.<a class="footnote-ref" id="fnref:11" href="#fn:11"><sup>11</sup></a><a class="footnote-ref" id="fnref:12" href="#fn:12"><sup>12</sup></a> </p> <h2 id="history">History</h2> <p>The US FDA approved nedosiran based on evidence from a clinical trial which included 29 participants with primary hyperoxaluria type 1.<a class="footnote-ref" id="fnref:13" href="#fn:13"><sup>13</sup></a> Nedosiran was evaluated in one clinical trial of 29 participants with primary hyperoxaluria type 1 who were nine years of age and older.<a class="footnote-ref" id="fnref:14" href="#fn:14"><sup>14</sup></a> Participants randomly received either nedosiran or placebo injections once a month for six months.<a class="footnote-ref" id="fnref:15" href="#fn:15"><sup>15</sup></a> Neither the participants nor the healthcare providers knew which treatment was being given.<a class="footnote-ref" id="fnref:16" href="#fn:16"><sup>16</sup></a> 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.<a class="footnote-ref" id="fnref:17" href="#fn:17"><sup>17</sup></a> </p> <h2 id="society-and-culture">Society and culture</h2> <h3>Legal status</h3> <p>The FDA granted the application for nedosiran <a href="/facts/Breakthrough_therapy/VYNK0Ve6">breakthrough therapy</a> and <a href="/facts/Orphan_drug/96judAS6">orphan drug</a> designations.<a class="footnote-ref" id="fnref:18" href="#fn:18"><sup>18</sup></a> Nedosiran was approved for medical use in the United States in September 2023.<a class="footnote-ref" id="fnref:19" href="#fn:19"><sup>19</sup></a><a class="footnote-ref" id="fnref:20" href="#fn:20"><sup>20</sup></a> </p> <h3>Names</h3> <p>Nedosiran is the <a href="/facts/International_nonproprietary_name/wYL655x6">international nonproprietary name</a>.<a class="footnote-ref" id="fnref:21" href="#fn:21"><sup>21</sup></a> </p> <h2 id="further-reading">Further reading</h2> <ul><li>Forbes TA, Brown BD, Lai C (June 2022). <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9291495">"Therapeutic RNA interference: A novel approach to the treatment of primary hyperoxaluria"</a>. <i>British Journal of Clinical Pharmacology</i>. 88 (6): 2525–2538. <a href="/facts/Doi_(identifier)/muM9Etpq">doi</a>:<a href="https://doi.org/10.1111%2Fbcp.14925">10.1111/bcp.14925</a>. <a href="/facts/PMC_(identifier)/dX1zMt71">PMC</a> <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9291495">9291495</a>. <a href="/facts/PMID_(identifier)/JlHAvMHt">PMID</a> <a href="https://pubmed.ncbi.nlm.nih.gov/34022071">34022071</a>.</li> <li>Liu A, Zhao J, Shah M, Migliorati JM, Tawfik SM, Bahal R, et al. (November 2022). <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9667536">"Nedosiran, a Candidate siRNA Drug for the Treatment of Primary Hyperoxaluria: Design, Development, and Clinical Studies"</a>. <i>ACS Pharmacology & Translational Science</i>. 5 (11): 1007–1016. <a href="/facts/Doi_(identifier)/muM9Etpq">doi</a>:<a href="https://doi.org/10.1021%2Facsptsci.2c00110">10.1021/acsptsci.2c00110</a>. <a href="/facts/PMC_(identifier)/dX1zMt71">PMC</a> <a href="https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9667536">9667536</a>. <a href="/facts/PMID_(identifier)/JlHAvMHt">PMID</a> <a href="https://pubmed.ncbi.nlm.nih.gov/36407951">36407951</a>.</li></ul> <h2 id="external-links">External links</h2> <ul><li>Clinical trial number <i><a href="https://www.clinicaltrials.gov/show/NCT03847909">NCT03847909</a></i> for "A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2 (PHYOX2)" at <a href="/facts/ClinicalTrials.gov/X6WVgItm">ClinicalTrials.gov</a></li> <li>Clinical trial number <i><a href="https://www.clinicaltrials.gov/show/NCT04042402">NCT04042402</a></i> for "Long Term Extension Study in Patients With Primary Hyperoxaluria (PHYOX3)" at <a href="/facts/ClinicalTrials.gov/X6WVgItm">ClinicalTrials.gov</a></li></ul> <h2 id="references">References</h2> <ol> <li id="fn:1"><p>"Rivfloza (nedosiran) injection, for subcutaneous use" (PDF). Archived (PDF) from the original on 1 October 2023. Retrieved 1 October 2023. <a href="https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf" target="_blank">https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf</a> <a href="#fnref:1" class="footnote-back-ref">↩</a></p></li> <li id="fn:2"><p>"Rivfloza (nedosiran) injection, for subcutaneous use" (PDF). Archived (PDF) from the original on 1 October 2023. Retrieved 1 October 2023. <a href="https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf" target="_blank">https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf</a> <a href="#fnref:2" class="footnote-back-ref">↩</a></p></li> <li id="fn:3"><p>"Nedosiran". Dicerna Pharmaceuticals. 17 August 2021. Archived from the original on 26 July 2023. Retrieved 1 October 2023. <a href="https://dicerna.com/pipeline/nedosiran/" target="_blank">https://dicerna.com/pipeline/nedosiran/</a> <a href="#fnref:3" class="footnote-back-ref">↩</a></p></li> <li id="fn:4"><p>"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. <a href="https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-rivfloza" target="_blank">https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-rivfloza</a> <a href="#fnref:4" class="footnote-back-ref">↩</a></p></li> <li id="fn:5"><p>"Rivfloza (nedosiran) injection, for subcutaneous use" (PDF). Archived (PDF) from the original on 1 October 2023. Retrieved 1 October 2023. <a href="https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf" target="_blank">https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf</a> <a href="#fnref:5" class="footnote-back-ref">↩</a></p></li> <li id="fn:6"><p>"Rivfloza: FDA-Approved Drugs". U.S. Food and Drug Administration (FDA). Archived from the original on 2 October 2023. Retrieved 1 October 2023. <a href="https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=215842" target="_blank">https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=215842</a> <a href="#fnref:6" class="footnote-back-ref">↩</a></p></li> <li id="fn:7"><p>"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 href="https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/215842Orig1s000TOC.cfm" target="_blank">https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/215842Orig1s000TOC.cfm</a> <a href="#fnref:7" class="footnote-back-ref">↩</a></p></li> <li id="fn:8"><p>"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. <a href="https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-rivfloza" target="_blank">https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-rivfloza</a> <a href="#fnref:8" class="footnote-back-ref">↩</a></p></li> <li id="fn:9"><p>New Drug Therapy Approvals 2023 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2024. Archived from the original on 10 January 2024. 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Retrieved 1 October 2023. <a href="https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf" target="_blank">https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf</a> <a href="#fnref:11" class="footnote-back-ref">↩</a></p></li> <li id="fn:12"><p>"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. <a href="https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-rivfloza" target="_blank">https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-rivfloza</a> <a href="#fnref:12" class="footnote-back-ref">↩</a></p></li> <li id="fn:13"><p>"Drug Trials Snapshots: Rivfloza". U.S. Food and Drug Administration (FDA). 29 September 2023. Retrieved 15 July 2024. 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Retrieved 1 October 2023. <a href="https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf" target="_blank">https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/215842s000lbl.pdf</a> <a href="#fnref:19" class="footnote-back-ref">↩</a></p></li> <li id="fn:20"><p>"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. <a href="https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-rivfloza" target="_blank">https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-rivfloza</a> <a href="#fnref:20" class="footnote-back-ref">↩</a></p></li> <li id="fn:21"><p>World Health Organization (2021). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 85". WHO Drug Information. 35 (1). hdl:10665/340684. <a href="/wiki/World_Health_Organization" target="_blank">/wiki/World_Health_Organization</a> <a href="#fnref:21" class="footnote-back-ref">↩</a></p></li> </ol>