Golodirsen

Summary

Golodirsen (CHEMBL4297762) is an approved oligonucleotide (ATC M09AX08); indicated across 2 conditions including duchenne muscular dystrophy and muscular dystrophy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Oligonucleotide
• ATC class: M09AX08
• Indications: 2 conditions
• Clinical trials: 6

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297762
Name	Golodirsen
Type	Oligonucleotide
Max phase	4
ATC	M09AX08

Also known as: Golodirsen, SRP-4053, Vyondys 53, GOLODIRSEN

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: 6.

Phase distribution

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

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04708314	PHASE4	TERMINATED	An Open-Label Study of Golodirsen in Non-Ambulant Patients With Duchenne Muscular Dystrophy
NCT02500381	PHASE3	COMPLETED	Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
NCT03532542	PHASE3	TERMINATED	An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
NCT02310906	PHASE1/PHASE2	COMPLETED	Phase I/II Study of SRP-4053 in DMD Patients
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).