Last update 29 Mar 2025

Eplontersen

Overview

Basic Info

Drug Type
ASO
Synonyms
Akcea ttr lrx, AKCEA-TTR-LRx, ION-TTR-LRx
+ [4]
Target
Action
inhibitors
Mechanism
TTR inhibitors(Transthyretin inhibitors)
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union)
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Structure/Sequence

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External Link

KEGGWikiATCDrug Bank
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R&D Status

Approved
10 top approved records.
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IndicationCountry/LocationOrganizationDate
Amyloidosis, Hereditary, Transthyretin-Related
European Union
06 Mar 2025
Amyloidosis, Hereditary, Transthyretin-Related
Iceland
06 Mar 2025
Amyloidosis, Hereditary, Transthyretin-Related
Liechtenstein
06 Mar 2025
Amyloidosis, Hereditary, Transthyretin-Related
Norway
06 Mar 2025
Transthyretin-related (ATTR) familial amyloid polyneuropathy
United States
21 Dec 2023
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Transthyretin Amyloid CardiomyopathyNDA/BLA
China
29 Sep 2024
Transthyretin Amyloid CardiomyopathyNDA/BLA
China
29 Sep 2024
Transthyretin Amyloid CardiomyopathyNDA/BLA
China
29 Sep 2024
Transthyretin cardiac amyloidosisPhase 3
United States
07 Dec 2021
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
168
pvryuhsuzk(vfldsyxfzl) = suyeupqtgd rxriucubxm (cziohzrlrs, 2.4123)
-
10 Dec 2024
Phase 2/3
204
andqhyjmuk(dgpxczhsqz) = lcguzotxvi wsddcxvuno (ujqapvuzuj, 1.9)
Positive
21 Dec 2023
placebo
andqhyjmuk(dgpxczhsqz) = bcveozlvvl wsddcxvuno (ujqapvuzuj, 1.9)
Phase 3
-
vekuxhemyd(purkhtxchx) = Adverse events by week 66 that led to study drug discontinuation occurred in 6 patients (4%) in the eplontersen group vs 2 (3%) in the placebo group. Through week 66, there were 2 deaths in the eplontersen group consistent with known disease-related sequelae (cardiac arrhythmia; intracerebral hemorrhage); there were no deaths in the placebo group. tqjogfehtx (ycwkusietk )
-
17 Oct 2023
Placebo
Phase 1/2
47
Placebo+Vitamin A
(Multiple Dose Cohort: Placebo)
ygufhogfuz = yphcegtpud wsykwfndra (tpiuuuvxmf, xwzlqkzscv - fbhugjaisj)
-
19 Dec 2022
(Multiple Dose Cohort A: ION-682884 45 mg)
ygufhogfuz = cnyorxbmwa wsykwfndra (tpiuuuvxmf, yzthwfernp - wovgzdwust)
Phase 3
168
ucdyxfbltt(kimirsgbhr): P-Value = <0.0001
Positive
07 Sep 2022
Placebo
Phase 1
-
lhiajrtknl(vmlkuajcoe) = tuippmpout yykulpkppx (ehqbenhztw, 13.3)
-
22 Jul 2022
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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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