Volanesorsen

Summary

Volanesorsen (CHEMBL3544994) is an approved oligonucleotide (ATC C10AX18); indicated across 3 conditions including cardiovascular disorder and hypertriglyceridemia.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Oligonucleotide
• ATC class: C10AX18
• Indications: 3 conditions
• Clinical trials: 7

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3544994
Name	Volanesorsen
Type	Oligonucleotide
Max phase	4
ATC	C10AX18

Also known as: ISIS 304801, ISIS 304801 FREE ACID, ISIS-304801, Volanesorsen, VOLANESORSEN

Parent form; salt/anhydrous children: CHEMBL3544989

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

3 indications (1 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
cardiovascular disorder	4	MONDO:0004995	EFO:0000319
hypertriglyceridemia	3	MONDO:0005347	EFO:0004211
familial partial lipodystrophy	2	MONDO:0020088	MONDO:0020088

Clinical trials

Total trials: 7.

Phase distribution

Phase	Trials
PHASE3	3
PHASE2/PHASE3	1
PHASE2	1
PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02211209	PHASE3	COMPLETED	The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome
NCT02300233	PHASE3	COMPLETED	The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With Hypertriglyceridemia
NCT02527343	PHASE2/PHASE3	TERMINATED	The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy
NCT02658175	PHASE3	COMPLETED	The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome
NCT02639286	PHASE2	COMPLETED	Efficacy, Safety and Tolerability of ISIS 304801 in People With Partial Lipodystrophy With an Open-Label Extension
NCT02910635	PHASE1	COMPLETED	A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers
NCT03544060	Not specified	NO_LONGER_AVAILABLE	Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).