Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as Fazisiran Injection
Fazisiran · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Fazirsiran 25 mg DB/200 mg OL | EXPERIMENTAL | Double-blind (DB) Period: Participants with no fibrosis: Administered on Day 1 and Week 4. Participants with fibrosis: Administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses. Open-label (OL) Period: Participants with fibrosis at Screening who received double-blind (DB) fazirsiran 25 mg and who completed the post-dose liver biopsy at Week 48 (or Week 72 or Week 96) entered the open-label phase and received fazirsiran 200 mg every 12 weeks for the duration of the study. |
| Fazirsiran 100 mg DB/200 mg OL | EXPERIMENTAL | DB Period: Participants with no fibrosis: Fazirsiran 100 mg administered on Day 1 and Week 4. Participants with fibrosis: Fazirsiran 100 mg administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses. OL Period: Participants with fibrosis at Screening who received double-blind (DB) fazirsiran 100 mg and who completed the post-dose liver biopsy at Week 48 (or Week 72 or Week 96) entered the open-label phase and received fazirsiran 200 mg every 12 weeks for the duration of the study. |
| Fazirsiran 200 mg DB/200 mg OL | EXPERIMENTAL | DB Period: Participants with no fibrosis: Fazirsiran 200 mg administered on Day 1 and Week 4. Participants with fibrosis: Fazirsiran 200 mg administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses. OL Period: Participants with fibrosis at Screening who received double-blind (DB) fazirsiran 200 mg and who completed the post-dose liver biopsy at Week 48 (or Week 72 or Week 96) entered the open-label phase and received fazirsiran 200 mg every 12 weeks for the duration of the study |
| Placebo DB / Fazirsiran 200 mg OL | PLACEBO_COMPARATOR | DB Period: Participants with no fibrosis: Placebo administered on Day 1 and Week 4. Participants with fibrosis: Placebo administered on Day 1, Week 4, and Week 16, then every 12 weeks up to 18 total doses. OL Period: Participants with fibrosis at Screening who received double-blind (DB) placebo and who completed the post-dose liver biopsy at Week 48 (or Week 72 or Week 96) entered the open-label phase and received fazirsiran 200 mg every 12 weeks for the duration of the study. |
| Name | Type | Description |
|---|---|---|
| Fazisiran Injection | DRUG | solution for subcutaneous (sc) injection |
| Placebo | OTHER | sterile normal saline (0.9% NaCl), calculated to match active comparator, for sc injection |
Inclusion Criteria: * Diagnosis of AATD * Liver biopsy at Screening indicating liver fibrosis (score less than F4); a patient with no fibrosis may participate based on a previous biopsy conducted within one year * Women of childbearing potential must have a negative pregnancy test, cannot be breast...
Fazisiran is an investigational RNA therapy being developed for the treatment of Alpha 1-Antitrypsin Deficiency, a rare genetic disorder. It is designed to reduce the production of the abnormal alpha-1 antitrypsin protein that causes liver disease in affected patients. Fazisiran is not yet approved for any indication.
Fazisiran is a small interfering RNA (siRNA) therapeutic, part of the -siran drug class. It works by targeting and degrading the messenger RNA that encodes alpha-1 antitrypsin, thereby reducing the synthesis of the mutant protein in the liver. This mechanism aims to lessen the accumulation of toxic protein that drives liver injury in Alpha 1-Antitrypsin Deficiency.
Fazisiran is being developed by Arrowhead Pharmaceuticals, Inc., a biopharmaceutical company focused on RNA interference therapeutics. Arrowhead trades on the Nasdaq under the ticker symbol ARWR. The company is responsible for the clinical development of Fazisiran for Alpha 1-Antitrypsin Deficiency.
Fazisiran is in Phase 2 clinical development. It has completed one Phase 2 trial and is not currently listed as active in any ongoing trials. The drug remains investigational and has not been approved by the FDA or any other regulatory agency for commercial use.
Fazisiran has been studied in a completed Phase 2 trial, NCT03945292, titled "Safety, Tolerability and Pharmacodynamic Effect of Fazisiran (TAK-999, ARO-AAT)." The trial enrolled 40 participants with Alpha 1-Antitrypsin Deficiency across the United States, Germany, Italy, Netherlands, Portugal, and Spain. It was randomized, double-blind, and placebo-controlled.
Yes, Fazisiran Injection is another name for Fazisiran. The drug is also known by the research codes TAK-999 and ARO-AAT, which appear in its clinical trial identifier. All these names refer to the same investigational siRNA therapy for Alpha 1-Antitrypsin Deficiency.