Olezarsen
Highest phase reached
Phase 3
Drug type
Oligonucleotide
- Listing at source
- Listed
- Highest phase reached
- Phase 3
- Drug type
- Oligonucleotide
- Drug name
- Olezarsen
- akcea apociii lrx
- Akcea-apociii-lrx
- AKCEA-APOCIII-LRx
- dna d p thio 2 o 2 methoxyethyl ra 2 o 2 methoxyethyl rg 2 o 2 methoxyethyl m5rc 2 o 2 methoxyethyl m5ru 2 o 2 methoxyethyl m5ru m5c t t g t m5c m5c a g m5c 2 o 2 methoxyethyl m5ru 2 o 2 methoxyethyl m5ru 2 o 2 methoxyethyl m5ru 2 o 2 methoxyethyl ra 2 o 2 methoxyethyl m5ru 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(P-THIO)((2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))RG-(2'-O-(2-METHOXYETHYL))M5RC-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))M5RU-M5C-T-T-G-T-M5C-M5C-A-G-M5C-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))M5RU-(2'-O-(2-METHOXYETHYL))RA-(2'-O-(2-METHOXYETHYL))M5RU), 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)
- ionis apociii lrx
- IONIS- APOCIII-LRx
- IONIS-APOCIII-LRX
- isis 678354
- ISIS 678354
- isis apociii lrx
- ISIS-678354
- ISIS-APOCIII-LRX
- olezarsen
- Olezarsen
- olezarsen [INN]
- OLEZARSEN [USAN]
- Olezarsen [WHO-DD]
- olezarsen inn
- olezarsen usan
- olezarsen who dd
- ATC classification codes
- C10AX
- CAS registry numbers
- 2097587-83-0
- ChEMBL ID
- CHEMBL4650438
- RxNorm IDs (RxCUI)
- 2701418
- FDA ingredient codes (UNII)
- S3RS2SA30L
- Status
- Authorised
- Status date
- 17 Sept 2025
- Authorisation date
- 17 Sept 2025
- Approved indications
- Tryngolza is indicated as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS).
- Product number
- EMEA/H/C/006477
- Authorisation holder
- Swedish Orphan Biovitrum AB (publ)
- 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
- Hyperlipoproteinemia Type I
European medicines record
- Patents
- 0
- Designations
- 5
- First in class
- No
- Fast track
- Yes
- Breakthrough designation
- Yes
- Orphan designation
- No
- Priority review
- Yes
- Orphan exclusivity
- No
- Accelerated approval
- No
- European Union (EMA)
- Authorised
- Pharmacology name
- olezarsen
- Type
- Nucleic acid
- Drug name stem
- -rsen
- Approval recorded by Guide to Pharmacology
- Yes
- Mechanism
- Apolipoprotein C-III mRNA antisense inhibitor
- Action type
- ANTISENSE_INHIBITOR
- Target
- Apolipoprotein C-III mRNA
- Protein identifier
- ENSG00000110245
- Mechanism
- Apolipoprotein C-III mRNA antisense inhibitor
- Action type
- Inhibitor
- Target
- Apolipoprotein C-III mRNA
- Protein identifier
- ENSG00000110245
Open Targets mechanisms
- Mechanism
- Apolipoprotein C-III mRNA antisense inhibitor
- Action type
- ANTISENSE INHIBITOR
- Target
- Apolipoprotein C-III mRNA
- Condition
- Cardiovascular Diseases
- Highest recorded phase
- 1
- Condition identifier
- EFO:0000319
- Subject identifier
- D002318
- Condition
- Hypertriglyceridemia
- Highest recorded phase
- 3
- Condition identifier
- EFO:0004211
- Subject identifier
- D015228
- Condition identifier
- EFO_0000319
- Highest recorded phase
- 2
- Condition identifier
- EFO_0004211
- Highest recorded phase
- 3
- Condition identifier
- MONDO_0004982
- Highest recorded phase
- 3
- Condition identifier
- MONDO_0004995
- Highest recorded phase
- 2
- Condition identifier
- MONDO_0005347
- Highest recorded phase
- 3
- Condition identifier
- MONDO_0009387
- Condition identifier
- MONDO_0009387
- Highest recorded phase
- 4
- Condition identifier
- MONDO_0018637
- Condition identifier
- MONDO_0018637
- Highest recorded phase
- 4
- NCT05079919
- NCT05552326
- NCT05681351
- Prodrug
- No
- Chirality
- Single enantiomer
- Inorganic
- No
- Natural product
- No
Sequences
- Component
- Sequence
- Type
- Nucleic Acid
- Sequence
- (3'-5')R-A=G=C=U=U=d(C=T=T=G=T=C=C=A=G=C=)U=U=U=A=U
Related records
Trials (14)
- A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult Participants
- A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
- A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
- A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Adults With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease (Established or at Increased Risk for), and/or With Severe Hypertriglyceridemia
- A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)
- A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia
- A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia
- A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia
- A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants
- CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
- Mechanistic Study of ISIS ISIS-APOCIIIRX in Type 2 Diabetes
- Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
- Safety, Tolerability, PK, and Pharmacodynamics(PD) of IONIS APOCIII-LRx in Healthy Volunteers With Elevated Triglycerides
- Study of ISIS 678354 (AKCEA-APOCIII-LRx) in Participants With Hypertriglyceridemia and Established Cardiovascular Disease (CVD)
Approved for (2)
- Familial chylomicronemia syndrome
- Familial lipoprotein lipase deficiency
In development for (3)
- Cardiovascular disorder Phase 1
- Hypertriglyceridemia Phase 3
- Pancreatitis Phase 3
Targets (1)
- APOC3 Apolipoprotein C-III mRNA antisense inhibitor