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Fazirsiran Injection
Fazirsiran Injection is a RNAi therapeutic (siRNA) Small molecule drug developed by Takeda. It is currently in Phase 3 development for Transfusion-dependent beta-thalassemia, Iron overload disorders. Also known as: TAK-999, ARO-AAT, ADS-001, TAK-999, ARO-AAT, ADS-001.
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.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Rare-disease pathway favourability
+5.0pp
Rare-disease drugs benefit from FDA Orphan Drug Act, smaller pivotal trials, and more flexible endpoints. Approval rates run ~5pp above baseline. -
Big-pharma sponsor
+3.0pp
Takeda is a top-20 pharma sponsor — historical approval rates run ~3pp above average due to scale, regulatory experience, and trial-design quality.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
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
Frequently asked questions about Fazirsiran Injection
What is Fazirsiran Injection?
How does Fazirsiran Injection work?
What is Fazirsiran Injection used for?
Who makes Fazirsiran Injection?
Is Fazirsiran Injection also known as anything else?
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What does Fazirsiran Injection target?
Related
- Drug class: All RNAi therapeutic (siRNA) drugs
- Target: All drugs targeting ALAS2 (aminolevulinic acid synthase 2)
- Manufacturer: Takeda — full pipeline
- Therapeutic area: All drugs in Hematology / Rare Genetic Disorders
- Indication: Drugs for Transfusion-dependent beta-thalassemia
- Indication: Drugs for Iron overload disorders
- Also known as: TAK-999, ARO-AAT, ADS-001, TAK-999, ARO-AAT, ADS-001
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing