Drugs / Eplontersen
Eplontersen
also known as
ION-682884 · AKCEA-TTR-LRx · ION-682884 FREE ACID · Ion-ttr-lrx · IONIS-TTR-LRx · ISIS-682884 FREE ACID
Antisense oligonucleotide targets TTR
Regulatory milestones approvals, filings & regulatory actions · 6 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed ATTRV30M amyloidosis 3 milestones | |||||
| Filed | Other | — | ATTRV30M amyloidosis | 2023-11-02 | “Eplontersen marketing applications accepted for review in the EU and Canada; potential U.S. approval in December 2023” ionis.com ↗ |
| Filed | EU (EMA) | — | ATTRV30M amyloidosis | 2023-11-02 | “Eplontersen marketing applications accepted for review in the EU and Canada; potential U.S. approval in December 2023” ionis.com ↗ |
| Filed | US (FDA) | — | ATTRV30M amyloidosis | 2023-02-22 | “Last month, the US Food and Drug Administration accepted a New Drug Application for eplontersen for the treatment of ATTRv-PN.” astrazeneca.com ↗ “accepted for review a New Drug Application (NDA) for eplontersen” ionis.com ↗ “Eplontersen NDA submitted to FDA” ionis.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansion | US (FDA) | WAINUA (AUTOINJECTOR) | — | 2026-04-15 | fda.gov ↗ |
| Approved | EU (EMA) | Wainzua | — | 2025-03-06 | europa.eu ↗ |
| Approved | US (FDA) | WAINUA (AUTOINJECTOR) | — | 2023-12-21 | fda.gov ↗ |
Trials 9
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 12 trials | ||||||
| Phase 1 | NCT06527755 | Aug → Sep 2024 | — | AstraZeneca | Completed | No outcome recorded |
| Phase 1 | NCT04302064 | Apr → Sep 2020 | — | Ionis Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT03728634 | Dec 2018 → Feb 2020 | hereditary amyloidosis | Ionis Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT04843020 | Jun 2021 → May 2025 | amyloidosis | Brigham and Women's Hospital | Withdrawn | No outcome recorded |
| Phase 35 trials · 1 met primary · 1 missed | ||||||
| Phase 3 | NCT06194825 | Dec 2023 → May 2026 overdue | amyloidosis | AstraZeneca | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05667493 | Nov 2022 → Aug 2029 expected | amyloidosis | Ionis Pharmaceuticals, Inc. | Enrolling by invitation | No outcome recorded |
| Phase 3 | NCT05071300 | Jan 2022 → Aug 2029 expected | familial amyloid neuropathy | Ionis Pharmaceuticals, Inc. | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04136171 | Mar 2020 → Apr 2026 overdue | amyloidosis | Ionis Pharmaceuticals, Inc. | Active not recruiting | Missed primary |
| Phase 3 | NCT04136184 | Dec 2019 → Apr 2023 | familial amyloid neuropathy | Ionis Pharmaceuticals, Inc. | Completed | Met primary |
| Comparator or background therapy1 trial | ||||||
| Phase 2 | NCT07608354 Background | Jun 2026 → Feb 2029 expected | amyloidosis | AstraZeneca | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 20
| Date | Issuer | Release |
|---|---|---|
| 2026-07-09 | AstraZeneca | Results Update on CARDIO-TTRansform Phase III trial for Wainua (eplontersen) in adults with transthyretin-mediated amyloid cardiomyopathy astrazeneca.com ↗
In a prespecified subgroup analysis of patients treated with Wainua monotherapy as compared to placebo, fewer primary composite events (CV mortality and recurrent CV events) were observed and this result was nominally significant. |
| 2026-07-09 | Ionis Pharmaceuticals | Results Update on CARDIO-TTRansform Phase 3 trial of eplontersen in adults with transthyretin-mediated amyloid cardiomyopathy ionis.com ↗
In this contemporary patient population treated with standard of care, including a majority on a stabilizer, adding eplontersen did not provide a statistically significant benefit. |
| 2025-03-10 | Ionis Pharmaceuticals | Regulatory WAINZUA (eplontersen) approved in the EU for the treatment of hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that Ionis and AstraZeneca’s WAINZUA (eplontersen) has been approved in the European Union (EU) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, commonly referred to as hATTR-PN or ATTRv-PN. |
| 2024-10-21 | AstraZeneca | Regulatory Wainzua (eplontersen) recommended for approval in the EU by CHMP for the treatment of adult patients with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis astrazeneca.com ↗
AstraZeneca and Ionis’ Wainzua (eplontersen) has been recommended for approval by the Committee for Medicinal Products for Human Use (CHMP) in the European Union (EU) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, commonly referred to as hATTR-PN or ATTRv-PN. |
| 2024-10-21 | Ionis Pharmaceuticals | Regulatory WAINZUA (eplontersen) recommended for approval in the EU by CHMP for the treatment of adults with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis ionis.com ↗ |
| 2024-05-23 | Ionis Pharmaceuticals | Results New data from the Phase 3 NEURO-TTRansform study evaluating WAINUA™ (eplontersen) to be presented at the 2024 International Symposium on Amyloidosis (ISA) ionis.com ↗
Results across NEURO-TTRansform subgroups show consistent benefit in neuropathy impairment and improved quality of life, regardless of patient segmentation |
| 2024-02-08 | Ionis Pharmaceuticals | Regulatory Eplontersen granted U.S. FDA Fast Track designation for patients with transthyretin-mediated amyloid cardiomyopathy ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Ionis and AstraZeneca's eplontersen, an investigational therapy for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults. |
| 2023-12-21 | AstraZeneca | Regulatory Wainua (eplontersen) granted first-ever regulatory approval in the US for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis astrazeneca.com ↗
AstraZeneca and Ionis’ Wainua (eplontersen) has been approved in the US for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. |
| 2023-12-21 | Ionis Pharmaceuticals | Regulatory WAINUA™ (eplontersen) granted regulatory approval in the U.S. for the treatment of adults with polyneuropathy of hereditary transthyretin-mediated amyloidosis ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has approved Ionis and AstraZeneca's WAINUA™ (eplontersen) for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN. |
| 2023-09-28 | Ionis Pharmaceuticals | Results Eplontersen Phase 3 results published in JAMA show consistent and sustained benefit ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that The Journal of the American Medical Association (JAMA) published positive results from the Phase 3 NEURO-TTRansform study of eplontersen, an investigational treatment for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). |
| 2023-07-10 | Ionis Pharmaceuticals | Results Eplontersen continued to show improvement in ATTRv-PN through 85 weeks ionis.com ↗
Ionis Pharmaceuticals (Nasdaq: IONS) today announced positive topline, 85-week data from the Phase 3 NEURO-TTRansform study in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). |
| 2023-04-24 | AstraZeneca | Results NEURO-TTRansform Phase III results presented at AAN showed eplontersen demonstrated consistent and sustained improvement in all measures of disease and quality of life through 66 weeks astrazeneca.com ↗
Detailed results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed AstraZeneca and Ionis’ eplontersen met all co-primary endpoints and secondary endpoints at 66 weeks versus an external placebo group. |
| 2023-04-24 | Ionis Pharmaceuticals | Results Eplontersen halted ATTRv-PN disease progression and improved neuropathy impairment and quality of life in Phase 3 study through 66 weeks ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that the Phase 3 NEURO-TTRansform study for AstraZeneca and Ionis' eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) met all co-primary endpoints and secondary endpoints at 66 weeks versus an external placebo group. |
| 2023-03-27 | AstraZeneca | Results Eplontersen demonstrated sustained benefit in Phase III trial for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) through 66 weeks astrazeneca.com ↗
Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed eplontersen met its co-primary endpoints through 66 weeks. |
| 2023-03-27 | Ionis Pharmaceuticals | Results Ionis reports positive topline 66-week results of eplontersen Phase 3 study for patients with ATTRv-PN ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from the 66-week analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca's eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN), a debilitating and potentially fatal disease that leads to peripheral nerve damage and motor disability. |
| 2023-03-07 | Ionis Pharmaceuticals | Regulatory Ionis announces FDA acceptance of New Drug Application for eplontersen for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) ionis.com ↗
Ionis Pharmaceuticals (Nasdaq: IONS) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review a New Drug Application (NDA) for eplontersen, an investigational antisense medicine for the treatment of people living with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). |
| 2022-09-07 | Ionis Pharmaceuticals | Results Ionis presents positive results from Phase 3 NEURO-TTRansform study at International Symposium on Amyloidosis ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today presented positive results from a planned 35-week interim analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca's eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). |
| 2022-06-21 | AstraZeneca | Results Eplontersen met co-primary and secondary endpoints in interim analysis of the NEURO-TTRansform Phase III trial for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) astrazeneca.com ↗
Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed AstraZeneca and Ionis’ eplontersen met its co-primary endpoints in a planned interim analysis at 35 weeks. |
| 2022-06-21 | Ionis Pharmaceuticals | Results Ionis announces eplontersen met co-primary and secondary endpoints in interim analysis of the Phase 3 NEURO-TTRansform study for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced positive topline results from a 35-week interim analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca's eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). |
| 2022-01-24 | Ionis Pharmaceuticals | Regulatory Ionis announces eplontersen receives orphan drug designation from U.S. FDA ionis.com ↗
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS), the leader in antisense therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to eplontersen, an investigational antisense medicine for the treatment of people living with transthyretin-mediated amyloidosis, a systemic, progressive and fatal condition. |
All press releases naming this drug 30 releases
Evidence & citations 11 cited values
Every value below carries the sentence it was read from. 12 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Eplontersen | “Eplontersen is a triantennary N-acetyl galactosamine (GalNAc3)-conjugated antisense oligonucleotide targeting TTR messenger ribonucleic acid (mRNA) to inhibit production of...” PMID 35869634 ↗ Aug 2022 |
| Known as | AKCEA-TTR-LRx | “A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary...” NCT05071300 ↗ |
| Known as | ION-682884 | “A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary...” NCT05071300 ↗3“An Open-Label Extension Study to Assess the Long-Term Safety of Eplontersen (ION-682884) in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)” NCT05667493 ↗ “ION-682884 in Patients With TTR Amyloid Cardiomyopathy” NCT04843020 ↗ |
| Known as | ION-682884 FREE ACID | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594334 ↗ |
| Known as | Ion-ttr-lrx | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594334 ↗ |
| Known as | IONIS-TTR-LRx | “A Study to Assess the Long-Term Safety and Efficacy of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Patients With Hereditary...” NCT05071300 ↗ |
| Known as | ISIS-682884 FREE ACID | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594334 ↗ |
| Action | Inhibit | “to inhibit production of both variant and wild-type TTR” PMID 35869634 ↗ Aug 2022 |
| Modality | Antisense oligonucleotide | “Eplontersen is a triantennary N-acetyl galactosamine (GalNAc3)-conjugated antisense oligonucleotide targeting TTR messenger ribonucleic acid (mRNA) to inhibit production of...” PMID 35869634 ↗ Aug 2022 |
| Route | Subcutaneous | “administered subcutaneously” PMID 42104840 ↗ May 2026 |
| Target | TTR | “targeting TTR messenger ribonucleic acid (mRNA)” PMID 35869634 ↗ Aug 2022 |