Phuturdex › Drugs › Fazirsiran
Drugs in development · updated 11 November 2026
Fazirsiran: clinical trials and development status
As of 11 November 2026 Fazirsiran is in 3 active clinical trials (recruiting or ongoing, phases I–III) for 1 disease. The most advanced phase is III.
Authorisation
- EU no (EMA)
- Italy not in the AIFA register
- Germany not available
- US no (FDA)
Sources: EMA, AIFA register (open data), FDA. For Germany only the EU centralised authorisation is shown: the national BfArM register is not one of our sources.
Map of sites in Europe with recruitment status, every trial and an email when something changes. Full access free until 31 March 2027, no card needed.
What it is
A formulation composed of the liver-targeted ligand N-acetylgalactosamine (GalNAc) conjugated to small-interfering RNA (siRNA) directed against wild-type and mutant alpha-1 antitrypsin, AAT and Z-AAT, that can potentially be used in the treatment of AAT deficiency (AATD)-associated liver disease. Upon administration of fazirsiran, the GalNAc moiety targets and binds with high affinity to asialoglycoprotein receptors (ASGPRs) expressed on hepatocytes. Once inside the cell, the siRNA binds to and silences AAT and Z-AAT messenger RNA (mRNA) and inhibits both the translation and expression of the AAT and Z-AAT proteins. This decreases the synthesis of the misfolded and poorly secreted Z-AAT protein, which lowers the accumulation of the inflammatory Z-AAT in hepatocytes. This may reduce inflammation and fibrosis, and prevent the progression of liver disease associated with AATD. SERPINA1 gene mutation in AATD leads to AAT deficiency and the production and accumulation of misfolded Z-AAT in the liver, triggering inflammation, fibrosis and hepatocellular injury.
Diseases studied
For each disease: the drug’s most advanced phase and active trials, with the reference trial on ClinicalTrials.gov.
| Disease | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| Alpha-1-antitrypsin deficiency | III | 3 | NCT05677971 |
Sites in Europe
49 sites of the active trials in 15 European countries, 47 recruiting.
- Austria6
- France6
- Germany6
- United Kingdom6
- Italy5
- Portugal5
- Spain4
- Belgium2
- and 7 more
Sites declared by the sponsors on ClinicalTrials.gov; region worked out by Phuturdex from the geographic point. The list of sites with city and recruitment status is in the app. See the sites →
Everything on Fazirsiran
In Phuturdex you get every trial with sponsors, countries and dates, development events, FDA and EMA approvals, patents and exclusivities, orphan designations and safety alerts, with PDF and Excel export. Free until 31 March 2027.
Open in Phuturdex →Source: Phuturdex analysis of ClinicalTrials.gov (U.S. National Library of Medicine), FDA and EMA data, updated 11 November 2026. Active trials = recruiting or ongoing, phases I–III. Diseases are those named by each trial (NLM MeSH terms). The estimated launch, when shown, refers to the United States and is a Phuturdex calculation described on the Method page, not official data: the drug may already be marketed in other countries.