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Olezarsen

Antisense Oligonucleotide

Also known as: akcea-apociii-lrx, ION-678354, ISIS 678354, olezarsen sodium, tryngolza

EMA approvedFDA approvedOrphan designation

Data last refreshed: (DrugCentral release 2024-11-01)

Key facts

Olezarsen is a drug in the Antisense Oligonucleotide class catalogued in DrugCentral. It was first approved by the FDA in 2024. DrugCentral lists 1 molecular target, including APOC3. Recorded indications include Familial chylomicronemia syndrome.

  • Drug class: Antisense Oligonucleotide; APOC-III-directed RNA Interaction.
  • Molecular targets: APOC3 (1 listed in DrugCentral).
  • Indications: Familial chylomicronemia syndrome.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (1)

  • Familial chylomicronemia syndrome

Mechanism & targets

Established pharmacologic classAntisense Oligonucleotide; APOC-III-directed RNA Interaction

Molecular targets (1)

TargetGeneActionActivity
apolipoprotein C-III (apoC-III) mRNAAPOC3otherIC50 6.74

Regulatory approvals (2)

DateAgencyMarketed asApplicantOrphan
2025-09-17EMA—Swedish Orphan Biovitrum ABYes
2024-12-19FDA—Ionis PharmaceuticalsYes

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

  • EMA label: LPL
  • FDA label: LPL

Chemical structure

Molecular formula—
Molecular weight—
CAS number—
InChIKey—
PubChem CID—

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Olezarsen used for?

DrugCentral lists the following indications for Olezarsen: Familial chylomicronemia syndrome.

How does Olezarsen work?

Its molecular targets in DrugCentral include APOC3.

When was Olezarsen first approved?

Olezarsen was first approved by the FDA in 2024.

What drug class does Olezarsen belong to?

Olezarsen is in the Antisense Oligonucleotide class.

Browse: all drugs starting with O · drug guides by category · protein targets A–Z.

Sources

How this page is built: methodology · editorial policy & corrections.

For research and educational use only — not medical advice. This page is compiled automatically from public databases. It is not written or reviewed by a clinician. Do not start, stop or change any treatment based on it; consult a qualified healthcare professional. Spotted an error? See our corrections policy or email hello@molforge.ai.

Quick facts

Status—
First approval2024-12-19
AgencyFDA
Targets1
Indications1
Formula—
DrugCentral ID5846
Interactive profile