Eplontersen
Highest phase reached
Phase 3
Drug type
Oligonucleotide
- Listing at source
- Listed
- Highest phase reached
- Phase 3
- Drug type
- Oligonucleotide
- Drug name
- Eplontersen
- 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.
- ATC classification codes
- N07XX21
Sources for recorded values
- CAS registry numbers
- 1637600-16-8
- ChEMBL ID
- CHEMBL4594334
- RxNorm IDs (RxCUI)
- 2671939
- FDA ingredient codes (UNII)
- 0GRZ0F5XJ6
- 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
- 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 name
- eplontersen
- Type
- Nucleic acid
- Drug name stem
- -rsen
- Approval recorded by Guide to Pharmacology
- Yes
- 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
- 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
- Condition identifier
- EFO_0004129
- Condition identifier
- EFO_1001875
- Condition identifier
- MONDO_0007100
- Highest recorded phase
- 4
- Condition identifier
- MONDO_0019065
- Condition identifier
- Orphanet_271861
- NCT04136184
- NCT05071300
- 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
- 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
- An Extension Study to Assess Long-Term Safety of Eplontersen in Adults With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)
- 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 Phase 1
- Familial amyloid neuropathy Phase 3
Targets (1)
- TTR Transthyretin mRNA antisense inhibitor