Donidalorsen

Summary

Donidalorsen (CHEMBL4650235) is a phase-3 clinical-stage oligonucleotide; indicated across 1 condition including hereditary angioedema.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligonucleotide
• Indications: 1 condition
• Clinical trials: 7

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4650235
Name	Donidalorsen
Type	Oligonucleotide
Max phase	3

Also known as: Donidalorsen, ISIS 721744 (free acid), ISIS-721744 FREE ACID, DONIDALORSEN

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

1 disease in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
hereditary angioedema	3	MONDO:0019623	MONDO:0019623

Clinical trials

Total trials: 7.

Phase distribution

Phase	Trials
PHASE3	3
PHASE2	2
PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05392114	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
NCT07298447	PHASE3	RECRUITING	Donidalorsen Treatment in Children With Hereditary Angioedema
NCT05139810	PHASE3	COMPLETED	OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
NCT04030598	PHASE2	COMPLETED	A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema
NCT04307381	PHASE2	COMPLETED	An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema
NCT03263507	PHASE1	COMPLETED	Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers
NCT06415448	Not specified	AVAILABLE	Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema

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).