Vutrisiran

Summary

Vutrisiran (CHEMBL4594511) is an approved oligonucleotide (ATC N07XX18); indicated across 3 conditions including familial amyloid neuropathy and polyneuropathy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Oligonucleotide
• ATC class: N07XX18
• Indications: 3 conditions
• Clinical trials: 5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4594511
Name	Vutrisiran
Type	Oligonucleotide
Max phase	4
ATC	N07XX18

Also known as: ALN-65492, ALN-TTRSC02, ALN-TTRSC02 FREE ACID, Votrisiran, Vutrisiran, VUTRISIRAN

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 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
familial amyloid neuropathy	4	MONDO:0007100	EFO:0004129
polyneuropathy	4	MONDO:0001824	EFO:0009562
amyloidosis	1	MONDO:0019065	EFO:1001875

Clinical trials

Total trials: 5.

Phase distribution

Phase	Trials
PHASE3	4
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04153149	PHASE3	ACTIVE_NOT_RECRUITING	HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
NCT06679946	PHASE3	ENROLLING_BY_INVITATION	A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
NCT07223203	PHASE3	RECRUITING	TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy
NCT03759379	PHASE3	COMPLETED	HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
NCT02797847	PHASE1	COMPLETED	A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy 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).