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Fazirsiran Injection
Fazirsiran is an RNA interference (RNAi) therapeutic that silences the ALAS2 gene to reduce heme synthesis and iron accumulation in patients with iron overload disorders.
Fazirsiran is an RNA interference (RNAi) therapeutic that silences the ALAS2 gene to reduce heme synthesis and iron accumulation in patients with iron overload disorders. Used for Transfusion-dependent beta-thalassemia, Iron overload disorders.
At a glance
| Generic name | Fazirsiran Injection |
|---|---|
| Also known as | TAK-999, ARO-AAT, ADS-001, TAK-999, ARO-AAT, ADS-001 |
| Sponsor | Takeda |
| Drug class | RNAi therapeutic (siRNA) |
| Target | ALAS2 (aminolevulinic acid synthase 2) |
| Modality | Small molecule |
| Therapeutic area | Hematology / Rare Genetic Disorders |
| Phase | Phase 3 |
Mechanism of action
Fazirsiran uses small interfering RNA (siRNA) technology to target and degrade ALAS2 messenger RNA, thereby decreasing aminolevulinic acid synthase 2 expression. This reduces heme production, which in turn decreases iron absorption and accumulation in tissues. The mechanism is designed to address the underlying pathophysiology of iron overload conditions by modulating the heme synthesis pathway.
Approved indications
- Transfusion-dependent beta-thalassemia
- Iron overload disorders
Common side effects
- Injection site reactions
- Transient liver enzyme elevations
- Platelet count changes
Key clinical trials
- A Study About Fazirsiran in People With and Without Liver Problems (PHASE1)
- 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) (PHASE3)
- 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 (PHASE3)
- Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT) (PHASE2)
- Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD) (PHASE2)
- An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Fazirsiran Injection CI brief — competitive landscape report
- Fazirsiran Injection updates RSS · CI watch RSS
- Takeda portfolio CI