Drisapersen

Summary

Drisapersen (CHEMBL2108735) is a phase-3 clinical-stage oligonucleotide (ATC M09AX04); indicated across 2 conditions including duchenne muscular dystrophy and muscular dystrophy.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligonucleotide
• ATC class: M09AX04
• Indications: 2 conditions
• Clinical trials: 8

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2108735
Name	Drisapersen
Type	Oligonucleotide
Max phase	3
ATC	M09AX04

Also known as: Drisapersen, Gsk 2402968a, GSK-2402968, GSK-2402968A, GSK2402968, GSK2402968A, H51AON23, PRO-051, PRO051, DRISAPERSEN

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 (0 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	3	MONDO:0010679	MONDO:0010679
muscular dystrophy	3	MONDO:0020121	MONDO:0020121

Clinical trials

Total trials: 8.

Phase distribution

Phase	Trials
PHASE3	4
PHASE2	3
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01254019	PHASE3	COMPLETED	A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
NCT01480245	PHASE3	TERMINATED	Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy
NCT01803412	PHASE3	TERMINATED	A Study of the Safety, Tolerability & Efficacy of Long-term Administration of Drisapersen in US & Canadian Subjects
NCT01890798	PHASE3	WITHDRAWN	Drisapersen Duchenne Muscular Dystrophy (DMD) Treatment Protocol
NCT01153932	PHASE2	COMPLETED	Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy
NCT01462292	PHASE2	COMPLETED	A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD)
NCT01910649	PHASE2	TERMINATED	A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration
NCT02636686	Not specified	NO_LONGER_AVAILABLE	Extension Study of Drisapersen in DMD Subjects

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).