Fitusiran

Summary

Fitusiran (CHEMBL4297754) is a phase-3 clinical-stage oligonucleotide; indicated across 3 conditions including hemophilia a and hemophilia b.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligonucleotide
• Indications: 3 conditions
• Clinical trials: 10

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297754
Name	Fitusiran
Type	Oligonucleotide
Max phase	3

Also known as: ALN-57213, Aln-at3sc, ALN-AT3SC, Fitusiran, FITUSIRAN

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 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
hemophilia A	3	MONDO:0010602	MONDO:0010602
hemophilia B	3	MONDO:0010604	MONDO:0010604

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

Clinical trials

Total trials: 10.

Phase distribution

Phase	Trials
PHASE3	7
PHASE4	1
PHASE1/PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06145373	PHASE4	RECRUITING	A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
NCT03754790	PHASE3	ACTIVE_NOT_RECRUITING	Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
NCT03974113	PHASE3	ACTIVE_NOT_RECRUITING	Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
NCT05662319	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
NCT07285460	PHASE3	RECRUITING	A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
NCT03417102	PHASE3	COMPLETED	A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
NCT03417245	PHASE3	COMPLETED	A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
NCT03549871	PHASE3	COMPLETED	A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis
NCT02554773	PHASE1/PHASE2	COMPLETED	An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B
NCT02035605	PHASE1	COMPLETED	A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients

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).