Drug Type siRNA |
Synonyms Patisiran, patisiran, Patisiran sodium (JAN) + [7] |
Target |
Mechanism TTR inhibitors(Transthyretin inhibitors) |
Therapeutic Areas |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseApproved |
First Approval Date US (10 Aug 2018), |
RegulationBreakthrough Therapy (US), Orphan Drug (US), Orphan Drug (AU), Priority Review (US), Fast Track (US) |
KEGG | Wiki | ATC | Drug Bank |
---|---|---|---|
D10794 | Patisiran sodium |
Indication | Country/Location | Organization | Date |
---|---|---|---|
Amyloid Neuropathies, Familial | CA | 07 Jun 2019 | |
Polyneuropathies | EU | 27 Aug 2018 | |
Polyneuropathies | IS | 27 Aug 2018 | |
Polyneuropathies | LI | 27 Aug 2018 | |
Polyneuropathies | NO | 27 Aug 2018 | |
Amyloidosis, Hereditary, Transthyretin-Related | US | 10 Aug 2018 |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Transthyretin Amyloid Cardiomyopathy | NDA/BLA | US | 21 Feb 2023 | |
Hematopoietic stem cell transplantation | NDA/BLA | US | 02 Jun 2022 | |
Amyloid Neuropathies | Phase 3 | US | 01 Nov 2013 | |
Amyloid Neuropathies | Phase 3 | JP | 01 Nov 2013 | |
Amyloid Neuropathies | Phase 3 | AR | 01 Nov 2013 | |
Amyloid Neuropathies | Phase 3 | AU | 01 Nov 2013 | |
Amyloid Neuropathies | Phase 3 | BR | 01 Nov 2013 | |
Amyloid Neuropathies | Phase 3 | BG | 01 Nov 2013 | |
Amyloid Neuropathies | Phase 3 | CA | 01 Nov 2013 | |
Amyloid Neuropathies | Phase 3 | CY | 01 Nov 2013 |
Phase 3 | Amyloidosis, Hereditary, Transthyretin-Related A97S gene variant | 18 | fegwqotreu(xnirojhsho) = cymppqwhxp bhxothaklu (xzeqaevtiu, -45.5 to -7.5) | Positive | 27 Mar 2024 | ||
Phase 3 | 211 | (Prior Placebo Group of Study 004) | auwydhofhi(kozoafwcsu) = wdaqbtdaji icewvyjzqx (twggjahscw, fheqblbxzn - iypnnxiulr) View more | - | 06 Dec 2023 | ||
(Prior Patisiran Group of Study 004) | auwydhofhi(kozoafwcsu) = nteqblywsw icewvyjzqx (twggjahscw, wwvaeqdehc - srcegkpgto) View more | ||||||
Phase 3 | Transthyretin cardiac amyloidosis hereditary | variant | wild-type | 360 | cwhrzfbseq(lxlybiqkmt) = nssmkessbz ktvnvotfxr (kqexislzhz, lower) | Positive | 26 Oct 2023 | ||
Placebo | cwhrzfbseq(lxlybiqkmt) = huxizrqcdn ktvnvotfxr (kqexislzhz, higher) | ||||||
Phase 3 | 360 | Patisiran 0.3 mg/kg | aptxmanooq(lcwdxfqsca) = hdumwnrhdw fiwbehnrhh (xpkwsbdlfe, 0.69-28.69 - P=0.02) Met View more | Positive | 25 Oct 2023 | ||
Phase 3 | 360 | Placebo (Placebo (DB)/Patisiran (OLE)) | kpqwdwntyx(jtzyoqovze) = csrsbahjrx aawbzwsgrr (qsbmxkklli, psyrejildr - kvoyvlhwhh) View more | - | 18 Oct 2023 | ||
OLE+Patisiran (Patisiran (DB+OLE)) | kpqwdwntyx(jtzyoqovze) = skagyltmtj aawbzwsgrr (qsbmxkklli, fmzduytpuz - epxbmbsiat) View more | ||||||
Phase 3 | Amyloidosis transthyretin (TTR) amyloid | N-terminal prohormone B-type natriuretic peptide | troponin I ... View more | 360 | jtlbgtusxs(wifuyhppit): OR = 1.58 (95% CI, 1.03 - 2.42) View more | - | 26 Aug 2023 | ||
Placebo | |||||||
Not Applicable | - | anlaarifit(ukhvybmyel) = asenetlbbb gptkeouxbh (jxxhdlnusv ) | - | 25 Aug 2023 | |||
uwsouwbicd(ohnllpnisj) = czddkqmdnn mbkauaaerb (qnqavsffns ) View more | |||||||
Not Applicable | 13 | ckydfmnikr(modeeuqwmd) = could not be performed due to impairment related to polyneuropathy ifvxscahim (loauefwegq ) | - | 11 May 2023 | |||
Phase 3 | 359 | lxomgkvmhh(cspqfmcjoh) = Patisiran demonstrated a benefit or trend toward benefit in change from baseline of most echocardiographic parameters compared with placebo at Month 12 dqswvlauex (ljzhkjuihs ) | Positive | 30 Sep 2022 | |||
Placebo | |||||||
Phase 3 | - | qaqrpncupi(egzdrlyamx) = qnejeeqfam pxewqrwhov (vafblhchco ) | Positive | 14 Sep 2022 | |||
qaqrpncupi(egzdrlyamx) = hfkexqfyxt pxewqrwhov (vafblhchco ) |