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Fazirsiran

Oligonucleotide (siRNA) · Gene silencing: alpha-1 antitrypsin (SERPINA1) · developed by Arrowhead Pharmaceuticals

Phase 2 Most advanced trial (none active)

ARWR Developer

3 Registered trials

What is Fazirsiran?

Fazirsiran is a GalNAc-conjugated siRNA targeting alpha-1 antitrypsin (AAT) mRNA. It silences both wild-type and Z-AAT expression in hepatocytes, reducing accumulation of misfolded Z-AAT protein and associated inflammation and fibrosis in alpha-1 antitrypsin deficiency-related liver disease.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as: ARO-AAT
Drug type: Oligonucleotide (siRNA)
Targets: alpha-1 antitrypsin (SERPINA1)
Mechanism: Gene silencing: alpha-1 antitrypsin (SERPINA1)
Pathways: protein synthesis inhibition, mRNA silencing
Therapy area: Gastroenterology, Respiratory
Developer: Arrowhead Pharmaceuticals (ARWR)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 2
UNII: V20IVC0OGQ

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT03946449	Phase 2	Alpha 1-Antitrypsin Deficiency	Completed	ARWR
NCT03945292	Phase 2	Alpha 1-Antitrypsin Deficiency	Completed	ARWR
NCT03362242	Phase 1	Alpha 1-Antitrypsin Deficiency	Completed	ARWR

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Alpha 1-Antitrypsin Deficiency

Other drugs acting on SERPINA1

ARC-AAT Phase 2 BEAM-302 Phase 1/2 NTLA-3001 Phase 1/2 WVE-006 Phase 1/2

More from Arrowhead Pharmaceuticals

Plozasiran FDA approved ARC-521 Phase 1 ARC-AAT Phase 2 ARO-033 Phase 1 ARO-ALK7 Phase 1/2 ARO-ANG 3 Phase 2 ARO-APOC3 Phase 2 ARO-ATXN2 Phase 1 ARO-C3 Phase 1/2 ARO-CFB Phase 1 ARO-DIMERPA Phase 1/2 ARO-ENaC Phase 1/2

Sources

Updated Oct 3, 2026. Something wrong or missing? Report it on the ARWR page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z