> Trialight public data. Index of pages: https://trialight.com/llms.txt
> Web page: https://trialight.com/data/asset/eplontersen-9180ea26-6471-4bf8-b8bc-f14ee872ae88
> Record changed: 2026-10-13

# Eplontersen

Highest phase reached

Phase 3

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4594334)

Drug type

Oligonucleotide

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4594334)

## Development status

**Listing at source**

Listed

**Highest phase reached**

Phase 3

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4594334)

## Names and chemistry

**Drug type**

Oligonucleotide

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4594334)

**Drug name**

Eplontersen

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4594334)

### Other names (28)

- akcea ttr lrx
- Akcea ttr lrx
- Akcea-ttr-lrx
- AKCEA-TTR-LRx
- all p ambo 5 o 6 5 tris 3 6 2 acetamido 2 deoxy beta d galactopyranosyloxy hexylamino 3 oxopropoxymethyl methyl amino 5 oxopentanamido hexoxy phospho 2 o 2 methoxyethyl 5 methyl p thiouridylyl 3 o 5 o 2 o 2 methoxyethyl 5 methylcytidylyl 3 o 5 o 2 o 2 methoxyethyl 5 methyluridylyl 3 o 5 o 2 o 2 methoxyethyl 5 methyluridylyl 3 o 5 o 2 o 2 methoxyethyl guanylyl 3 o 5 o 2 deoxy p thioguanylyl 3 o 5 o p thiothymidylyl 3 o 5 o p thiothymidylyl 3 o 5 o 2 deoxy p thioadenylyl 3 o 5 o 2 deoxy 5 methyl p thiocytidylyl 3 o 5 o 2 deoxy p thioadenylyl 3 o 5 o p thiothymidylyl 3 o 5 o 2 deoxy p thioguanylyl 3 o 5 o 2 deoxy p thioadenylyl 3 o 5 o 2 deoxy p thioadenylyl 3 o 5 o 2 o 2 methoxyethyl adenylyl 3 o 5 o 2 o 2 methoxyethyl 5 methyluridylyl 3 o 5 o 2 o 2 methoxyethyl 5 methyl p thiocytidylyl 3 o 5 o 2 o 2 methoxyethyl 5 methyl p thiocytidylyl 3 o 5 o 2 o 2 methoxyethyl 5 methylcytidine
- ALL-P-AMBO-5-O-(((6-(5-((TRIS(3-(6-(2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYLOXY)HEXYLAMINO)-3-OXOPROPOXYMETHYL))METHYL)AMINO-5-OXOPENTANAMIDO)HEXOXY))PHOSPHO)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLCYTIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)GUANYLYL-(3-O->5-O)-2-DEOXY-P-THIOGUANYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-2-DEOXY-P-THIOADENYLYL-(3-O->5-O)-2-DEOXY-5-METHYL-P-THIOCYTIDYLYL-(3-O->5-O)-2-DEOXY-P-THIOADENYLYL-(3-O->5-O)-P-THIOTHYMIDYLYL-(3-O->5-O)-2-DEOXY-P-THIOGUANYLYL-(3-O->5-O)-2-DEOXY-P-THIOADENYLYL-(3-O->5-O)-2-DEOXY-P-THIOADENYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)ADENYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLURIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOCYTIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYL-P-THIOCYTIDYLYL-(3-O->5-O)-2-O-(2-METHOXYETHYL)-5-METHYLCYTIDINE
- dna d 2 o 2 methoxyethyl m5ru sp 2 o 2 methoxyethyl m5rc 2 o 2 methoxyethyl m5ru 2 o 2 methoxyethyl m5ru 2 o 2 methoxyethyl rg g sp t sp t sp a sp m5c sp a sp t sp g sp a sp a sp 2 o 2 methoxyethyl ra 2 o 2 methoxyethyl m5ru 2 o 2 methoxyethyl m5rc sp 2 o 2 methoxyethyl m5rc sp 2 o 2 methoxyethyl m5rc 5 26 2 acetylamino 2 deoxy beta d galactopyranosyl oxy 14 14 bis 3 6 2 acetylamino 2 deoxy beta d galactopyranosyl oxy hexyl amino 3 oxopropoxy methyl 8 12 19 trioxo 16 oxa 7 13 20 triazahexacos 1 yl hydrogen phosphate
- DNA, D((2'-O-(2-METHOXYETHYL))M5RU-SP-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))RG-G-SP-T-SP-T-SP-A-SP-M5C-SP-A-SP-T-SP-G-SP-A-SP-A-SP-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))M5RC-SP-(2'-O-(2-METHOXYETHYL))M5RC), 5'-(26-((2-(ACETYLAMINO)-2-DEOXY-.BETA.-D-GALACTOPYRANOSYL)OXY)-14,14-BIS((3-((6-((2-(ACETYLAMINO)-2-DEOXY-.BETA.-D-GALACTOPYRANOSYL)OXY)HEXYL)AMINO)-3-OXOPROPOXY)METHYL)-8,12,19-TRIOXO-16-OXA-7,13,20-TRIAZAHEXACOS-1-YL HYDROGEN PHOSPHATE)
- eplontersen
- Eplontersen
- eplontersen \[INN\]
- Eplontersen \[MI\]
- EPLONTERSEN \[USAN\]
- Eplontersen \[WHO-DD\]
- eplontersen inn
- eplontersen mi
- eplontersen usan
- eplontersen who dd
- ion 682884
- ion 682884 free acid
- ion ttr lrx
- ION-682884
- ION-682884 FREE ACID
- Ion-ttr-lrx
- ionis ttr lrx

And 3 more, listed on the interactive page.

## Identifiers

**ATC classification codes**

- N07XX21

Sources for recorded values

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4594334)

**CAS registry numbers**

- 1637600-16-8

**ChEMBL ID**

CHEMBL4594334

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4594334)

**RxNorm IDs (RxCUI)**

- 2671939

**FDA ingredient codes (UNII)**

- 0GRZ0F5XJ6

## Regulatory information

### European Union

**Status**

Authorised

**Status date**

6 Mar 2025

**Authorisation date**

6 Mar 2025

**Approved indications**

Treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy.

**Product number**

EMEA/H/C/006295

**Authorisation holder**

AstraZeneca AB

**Priority medicines scheme**

No

**Generic**

No

**Biosimilar**

No

**Generic medicines**

0

**Biosimilar medicines**

0

**Advanced therapy**

No

**Conditional approval**

No

**Additional monitoring**

Yes

**Accelerated assessment**

No

**Exceptional circumstances**

No

#### Therapeutic areas

- Amyloid Neuropathies, Familial

European medicines record

Source: [www.ema.europa.eu](https://www.ema.europa.eu/en/medicines/human/EPAR/wainzua)

### Other regulatory facts

**Patents**

0

**Designations**

1

**First in class**

No

**Fast track**

No

**Breakthrough designation**

No

**Orphan designation**

No

**Priority review**

No

**Orphan exclusivity**

No

**Accelerated approval**

No

## Pharmacology

### Drug classification

**Pharmacology name**

eplontersen

**Type**

Nucleic acid

**Drug name stem**

\-rsen

**Approval recorded by Guide to Pharmacology**

Yes

### Mechanisms of action (2)

- **Mechanism**  
Transthyretin mRNA antisense inhibitor

**Action type**  
ANTISENSE\_INHIBITOR

**Target**  
Transthyretin mRNA

**Protein identifier**  
ENSG00000118271
- **Mechanism**  
Transthyretin mRNA antisense inhibitor

**Action type**  
Inhibitor

**Target**  
Transthyretin mRNA

**Protein identifier**  
ENSG00000118271

### Open Targets mechanisms

- **Mechanism**  
Transthyretin mRNA antisense inhibitor

**Action type**  
ANTISENSE INHIBITOR

**Target**  
Transthyretin mRNA

### Investigated indications (2)

- **Condition**  
Amyloid Neuropathies, Familial

**Highest recorded phase**  
3

**Condition identifier**  
EFO:0004129

**Subject identifier**  
D028227
- **Condition**  
Amyloidosis

**Highest recorded phase**  
1

**Condition identifier**  
EFO:1001875

**Subject identifier**  
D000686

### Open Targets indications (5)

- **Condition identifier**  
EFO\_0004129
- **Condition identifier**  
EFO\_1001875
- **Condition identifier**  
MONDO\_0007100

**Highest recorded phase**  
4
- **Condition identifier**  
MONDO\_0019065
- **Condition identifier**  
Orphanet\_271861

### Trial references (2)

- NCT04136184  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04136184)
- NCT05071300  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05071300)

## Chemical properties

**Prodrug**

No

**Chirality**

Single enantiomer

**Inorganic**

No

**Natural product**

No

### Sequences

- **Component**  
Sequence

**Type**  
Nucleic Acid

**Sequence**  
(3'-5')R1-U=C-U-U-G-d(G=T=T=A=C=A=T=G=A=A=)A-U-C=C=C

## National Drug Codes

- 0310-9400
- 0310-9420

## Related records

### Trials (10)

- 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 Transthyretin-Mediated Amyloid Polyneuropathy
- A Study to Assess the PK, PD, Safety and Tolerability of Eplontersen in Healthy Chinese Volunteers
- A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending and Multiple Doses of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Healthy Japanese Participants
- [A Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo for Adults Participants With ATTR-CM](https://trialight.com/data/trial/a-study-to-evaluate-the-efficacy-and-safety-of-concomitant-b3f29815-0f57-461a-9707-597acb769dc4.md)
- [An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)](https://trialight.com/data/trial/an-extension-study-to-assess-long-term-safety-of-eplontersen-591c383e-727e-459b-aacf-c9411087ae73.md)
- CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
- EPIC-ATTR: A Study to Evaluate the Effect of Eplontersen on the Transthyretin Reduction and Long-term Safety in Chinese Subjects With Transthyretin Amyloid Cardiomyopathy
- Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ).
- ION-682884 in Patients With TTR Amyloid Cardiomyopathy
- NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

### In development for (2)

- [Amyloidosis](https://trialight.com/data/condition/amyloidosis-b8b4cb9d-0eae-5851-8654-8d69fd195e67.md) Phase 1
- Familial amyloid neuropathy Phase 3

### Targets (1)

- [TTR](https://trialight.com/data/target/ttr-f983bfa0-69c4-59b3-857f-3a78a0800dc6.md) Transthyretin mRNA antisense inhibitor

---

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