Fazirsiran
drugOn this page
Also known as ADS-001ADS-001 FREE ACID
Summary
Fazirsiran (CHEMBL5095435) is a phase-3 clinical-stage oligonucleotide; indicated across 2 conditions including alpha 1-antitrypsin deficiency and liver disorder.
At a glance
- Status: Max clinical phase 3 (not approved)
- Modality: Oligonucleotide
- Indications: 2 conditions
- Clinical trials: 4
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL5095435 |
| Name | Fazirsiran |
| Type | Oligonucleotide |
| Max phase | 3 |
Also known as: ADS-001, ADS-001 FREE ACID, Fazirsiran, FAZIRSIRAN
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 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 |
|---|---|---|---|
| alpha 1-antitrypsin deficiency | 2 | MONDO:0013282 | Orphanet:60 |
| liver disorder | 1 | MONDO:0005154 | EFO:0001421 |
Clinical trials
Total trials: 4.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE3 | 3 |
| PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT05677971 | PHASE3 | RECRUITING | Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein |
| NCT05899673 | PHASE3 | ACTIVE_NOT_RECRUITING | An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease |
| NCT06165341 | PHASE3 | RECRUITING | Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis) |
| NCT05891158 | PHASE1 | COMPLETED | A Study About Fazirsiran in People With and Without Liver Problems |
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).
Related Atlas pages
No linked Atlas pages yet — the cross-entity mesh grows as the corpus expands.