Lumasiran

Summary

Lumasiran (CHEMBL4297779) is an approved oligonucleotide (ATC A16AX18); indicated across 3 conditions including primary hyperoxaluria and nephrolithiasis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Oligonucleotide
• ATC class: A16AX18
• Indications: 3 conditions
• Clinical trials: 8

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297779
Name	Lumasiran
Type	Oligonucleotide
Max phase	4
ATC	A16AX18

Also known as: AD-65585, ALN-65585, ALN-G01, ALN-GO1, Lumasiran, LUMASIRAN

Parent form; salt/anhydrous children: CHEMBL4650275

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 (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
primary hyperoxaluria	3	MONDO:0002474	MONDO:0002474
nephrolithiasis	2	MONDO:0008171	EFO:0004253

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 8.

Phase distribution

Phase	Trials
PHASE3	3
PHASE2	3
PHASE1/PHASE2	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03681184	PHASE3	COMPLETED	A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
NCT03905694	PHASE3	COMPLETED	A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
NCT04152200	PHASE3	COMPLETED	A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1
NCT06225544	PHASE2	RECRUITING	Lumasiran in Hyperoxalaemic Patients on Haemodialysis
NCT02706886	PHASE1/PHASE2	COMPLETED	Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
NCT03350451	PHASE2	COMPLETED	An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1
NCT05161936	PHASE2	TERMINATED	A Study to Evaluate Lumasiran in Adults With Recurrent Calcium Oxalate Kidney Stone Disease and Elevated Urinary Oxalate Levels
NCT04125472	Not specified	APPROVED_FOR_MARKETING	Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1

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).