Last update 27 Mar 2026

Patisiran sodium

Overview

Basic Info

Drug Type
siRNA
Synonyms
Patisiran, patisiran, Patisiran sodium (JAN)
+ [8]
Target
Action
inhibitors
Mechanism
TTR inhibitors(Transthyretin inhibitors)
Inactive Indication-
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
RegulationPriority Review (United States), Breakthrough Therapy (United States), Orphan Drug (United States), Orphan Drug (Australia), Fast Track (United States)
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Structure/Sequence

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External Link

R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Polyneuropathies
European Union
27 Aug 2018
Polyneuropathies
Iceland
27 Aug 2018
Polyneuropathies
Liechtenstein
27 Aug 2018
Polyneuropathies
Norway
27 Aug 2018
Amyloidosis, Hereditary, Transthyretin-Related
United States
10 Aug 2018
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Transthyretin Amyloid CardiomyopathyNDA/BLA
United States
21 Feb 2023
Amyloid Neuropathies, FamilialPhase 3
United States
16 Jul 2015
Amyloid Neuropathies, FamilialPhase 3
Japan
16 Jul 2015
Amyloid Neuropathies, FamilialPhase 3
Argentina
16 Jul 2015
Amyloid Neuropathies, FamilialPhase 3
Australia
16 Jul 2015
Amyloid Neuropathies, FamilialPhase 3
Brazil
16 Jul 2015
Amyloid Neuropathies, FamilialPhase 3
Bulgaria
16 Jul 2015
Amyloid Neuropathies, FamilialPhase 3
Canada
16 Jul 2015
Amyloid Neuropathies, FamilialPhase 3
Cyprus
16 Jul 2015
Amyloid Neuropathies, FamilialPhase 3
France
16 Jul 2015
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
18
Patisiran 0.3 mg/kg
juibpfukae(pthcssfpjf) = btagazliqr jctlcrdkgx (ssjpvvtrti, -45.5 to -7.5)
Positive
01 Sep 2024
Phase 3
211
(Prior Placebo Group of Study 004)
kzqerrmflm = jebbotwyry zccowbzyci (gmdmphqvcf, siswmcpwbr - rjqefixraw)
-
06 Dec 2023
(Prior Patisiran Group of Study 004)
kzqerrmflm = adtkalhziq zccowbzyci (gmdmphqvcf, xrxktgczuq - qtyodztrne)
Phase 3
Transthyretin Amyloid Cardiomyopathy
hereditary | variant | wild-type
360
xncqikgnce(ofiwcqinfv) = kbystovkid wawwrjgknv (ihtrfokaow, lower)
Positive
26 Oct 2023
Placebo
xncqikgnce(ofiwcqinfv) = qryhodwbak wawwrjgknv (ihtrfokaow, higher)
Phase 3
360
usoojvrwbe(ngwsiqrgaa) = bfbdokksib lncvchqkxf (tmotmlkkqp, 0.69-28.69 - P=0.02)
Met
Positive
25 Oct 2023
Phase 3
360
(Placebo (DB)/Patisiran (OLE))
ahqjkuazlk(xyrhsmngia) = bwxrhlkrwr jbqsypowgh (ugmvwntxwz, ujpwayoqxz - qlsyxvatks)
-
18 Oct 2023
(Patisiran (DB+OLE))
ahqjkuazlk(xyrhsmngia) = zbegxdfuqg jbqsypowgh (ugmvwntxwz, cwdhbtxetp - snsfohmpfn)
Phase 3
Amyloidosis
transthyretin (TTR) amyloid | N-terminal prohormone B-type natriuretic peptide | troponin I ...
360
ioqbeagavr(grugmdawgs): OR = 1.58 (95% CI, 1.03 - 2.42)
-
26 Aug 2023
Placebo
Phase 3
359
qgzwzpghiv(ifnhtghbsu) = Patisiran demonstrated a benefit or trend toward benefit in change from baseline of most echocardiographic parameters compared with placebo at Month 12 hxittejrqw (znlwxlmqsw )
Positive
30 Sep 2022
Placebo
Phase 3
-
poitzzveul(uyzwrzxagj) = stzkuoxvvl nwmmlofpsy (pnycmoalhl )
Positive
14 Sep 2022
poitzzveul(uyzwrzxagj) = kxwmsgjmrh nwmmlofpsy (pnycmoalhl )
Phase 3
360
rrvuyjixck(wdmtvwegih) = bwfymtvvxl uhflhlgclr (vohgfvqauy )
Positive
08 Sep 2022
Placebo
rrvuyjixck(wdmtvwegih) = cppgvixvoo uhflhlgclr (vohgfvqauy )
Phase 3
23
ijgstneent(bdbrnmgrcv) = ysrafngwsl hysdgzoput (ubvqnrypmn, 86.1 - 92.3)
Positive
25 Feb 2022
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Translational Medicine

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Deal

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Core Patent

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Clinical Trial

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Approval

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Regulation

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