Viltolarsen

Summary

Viltolarsen (CHEMBL4298062) is an approved oligonucleotide (ATC M09AX12); indicated across 2 conditions including duchenne muscular dystrophy and muscular dystrophy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Oligonucleotide
• ATC class: M09AX12
• Indications: 2 conditions
• Clinical trials: 8

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4298062
Name	Viltolarsen
Type	Oligonucleotide
Max phase	4
ATC	M09AX12

Also known as: NCNP-01, NS-065, Ns-065/ncnp-01, NS-065/NCNP-01, Viltepso, Viltolarsen, VILTOLARSEN

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

2 indications (2 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
Duchenne muscular dystrophy	4	MONDO:0010679	MONDO:0010679
muscular dystrophy	4	MONDO:0020121	MONDO:0020121

Clinical trials

Total trials: 8.

Phase distribution

Phase	Trials
PHASE2	3
PHASE3	2
PHASE4	1
PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04687020	PHASE4	ACTIVE_NOT_RECRUITING	Long-term Use of Viltolarsen in Boys With Duchenne Muscular Dystrophy in Clinical Practice (VILT-502)
NCT04060199	PHASE3	COMPLETED	Study to Assess the Efficacy and Safety of Viltolarsen in Ambulant Boys With DMD (RACER53)
NCT04768062	PHASE3	UNKNOWN	Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys With DMD (RACER53-X)
NCT02740972	PHASE2	COMPLETED	Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
NCT03167255	PHASE2	COMPLETED	Extension Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
NCT04956289	PHASE2	COMPLETED	Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
NCT02081625	PHASE1	COMPLETED	Exploratory Study of NS-065/NCNP-01 in DMD
NCT04337112	Not specified	APPROVED_FOR_MARKETING	The Expanded Access Use of Viltolarsen in Duchenne Muscular Dystrophy With Confirmed Exon 53 Amenable Mutation

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).