Eteplirsen

Summary

Eteplirsen (CHEMBL2108278) is an approved oligonucleotide (ATC M09AX06); indicated across 2 conditions including duchenne muscular dystrophy and muscular dystrophy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Oligonucleotide
• ATC class: M09AX06
• Indications: 2 conditions
• Clinical trials: 12

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2108278
Name	Eteplirsen
Type	Oligonucleotide
Max phase	4
ATC	M09AX06

Also known as: AVI-4658, Eteplirsen, Eteplirsen component of exondys 51, Exondys 51, ETEPLIRSEN

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: 12.

Phase distribution

Phase	Trials
PHASE2	7
PHASE3	2
PHASE1/PHASE2	2
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03992430	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
NCT02255552	PHASE3	COMPLETED	Study of Eteplirsen in DMD Patients
NCT00159250	PHASE1/PHASE2	COMPLETED	Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy
NCT00844597	PHASE1/PHASE2	COMPLETED	Dose-Ranging Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy (DMD) Patients
NCT01396239	PHASE2	COMPLETED	Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients
NCT01540409	PHASE2	COMPLETED	Efficacy, Safety, and Tolerability Rollover Study of Eteplirsen in Subjects With Duchenne Muscular Dystrophy
NCT02286947	PHASE2	COMPLETED	Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
NCT02420379	PHASE2	COMPLETED	Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy
NCT03218995	PHASE2	COMPLETED	Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
NCT03985878	PHASE2	TERMINATED	A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
NCT04179409	PHASE2	COMPLETED	A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.
NCT06606340	Not specified	ENROLLING_BY_INVITATION	A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice

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).