Drug Type ASO |
Synonyms Akcea ttr lrx, AKCEA-TTR-LRx, Eplontersen sodium + [6] |
Target |
Action inhibitors |
Mechanism TTR inhibitors(Transthyretin inhibitors) |
Therapeutic Areas |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
License Organization |
Drug Highest PhaseApproved |
First Approval Date United States (21 Dec 2023), |
RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Orphan Drug (Japan) |


| KEGG | Wiki | ATC | Drug Bank |
|---|---|---|---|
| - | - | - |
| Indication | Country/Location | Organization | Date |
|---|---|---|---|
| Amyloidosis, Hereditary, Transthyretin-Related | European Union | 10 Mar 2025 | |
| Amyloidosis, Hereditary, Transthyretin-Related | Iceland | 10 Mar 2025 | |
| Amyloidosis, Hereditary, Transthyretin-Related | Liechtenstein | 10 Mar 2025 | |
| Amyloidosis, Hereditary, Transthyretin-Related | Norway | 10 Mar 2025 | |
| Transthyretin-related (ATTR) familial amyloid polyneuropathy | United States | 21 Dec 2023 |
| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Transthyretin Amyloid Cardiomyopathy | Phase 3 | United States | 13 Mar 2020 | |
| Transthyretin Amyloid Cardiomyopathy | Phase 3 | Japan | 13 Mar 2020 | |
| Transthyretin Amyloid Cardiomyopathy | Phase 3 | Argentina | 13 Mar 2020 | |
| Transthyretin Amyloid Cardiomyopathy | Phase 3 | Australia | 13 Mar 2020 | |
| Transthyretin Amyloid Cardiomyopathy | Phase 3 | Austria | 13 Mar 2020 | |
| Transthyretin Amyloid Cardiomyopathy | Phase 3 | Belgium | 13 Mar 2020 | |
| Transthyretin Amyloid Cardiomyopathy | Phase 3 | Brazil | 13 Mar 2020 | |
| Transthyretin Amyloid Cardiomyopathy | Phase 3 | Canada | 13 Mar 2020 | |
| Transthyretin Amyloid Cardiomyopathy | Phase 3 | Czechia | 13 Mar 2020 | |
| Transthyretin Amyloid Cardiomyopathy | Phase 3 | Denmark | 13 Mar 2020 |
Phase 3 | 168 | jhuirztfdj(srfrrihvaq) = vwzidjqkza hstjztbpqp (dszljmaedm, 2.4123) View more | - | 10 Dec 2024 | |||
Phase 2/3 | 204 | ywcdxesfkt(qlrbwffzwr) = yhuftosfiq knmdmoephz (sbonrmaewn, 1.9) View more | Positive | 21 Dec 2023 | |||
placebo | ywcdxesfkt(qlrbwffzwr) = nzwpzzqcbg knmdmoephz (sbonrmaewn, 1.9) View more | ||||||
Phase 3 | - | mhvvmxpxkh(hveucnvojb) = 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. qxbiqgoxbj (mawpuoqkmt ) View more | - | 17 Oct 2023 | |||
Placebo | |||||||
Phase 1/2 | 47 | Placebo+Vitamin A (Multiple Dose Cohort: Placebo) | txfgpvjgcp = driwvwfxab nmljbmuakg (pwavzfvljm, bufyvihtod - fygxzzmddn) View more | - | 19 Dec 2022 | ||
(Multiple Dose Cohort A: ION-682884 45 mg) | txfgpvjgcp = fpgklvmacq nmljbmuakg (pwavzfvljm, gwnvkcrqzh - nvbxhvpqsc) View more | ||||||
Phase 3 | 168 | raoziuvvft(nibpxwxcwv): P-Value = <0.0001 View more | Positive | 07 Sep 2022 | |||
Placebo | |||||||
Phase 1 | - | twyawwupeb(ctvgdwxwhg) = bdcvhlxdys noghhomicw (smmcnfkyrn, 13.3) View more | - | 22 Jul 2022 |





