Givosiran

Summary

Givosiran (CHEMBL4297760) is a phase-3 clinical-stage oligonucleotide (ATC A16AX16); indicated across 3 conditions including hereditary coproporphyria and acute intermittent porphyria.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligonucleotide
• ATC class: A16AX16
• Indications: 3 conditions
• Clinical trials: 5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297760
Name	Givosiran
Type	Oligonucleotide
Max phase	3
ATC	A16AX16

Also known as: ALN-AS1, Givosiran, GIVOSIRAN

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

3 diseases 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 coproporphyria	3	MONDO:0007369	MONDO:0007369
acute intermittent porphyria	3	MONDO:0008294	MONDO:0008294
hepatic porphyria	3	MONDO:0002520	Orphanet:95157

Clinical trials

Total trials: 5.

Phase distribution

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

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03338816	PHASE3	COMPLETED	ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
NCT02949830	PHASE1/PHASE2	COMPLETED	A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
NCT02452372	PHASE1	COMPLETED	A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)
NCT03505853	PHASE1	COMPLETED	A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)
NCT04056481	Not specified	APPROVED_FOR_MARKETING	Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

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).