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ALN-AT3SC
ALN-AT3SC is a RNA interference therapeutic Small molecule drug developed by Sanofi. It is currently in Phase 1 development.
ALN-AT3SC uses RNA interference to silence the antithrombin gene, reducing antithrombin levels to promote blood clotting in bleeding disorders.
ALN-AT3SC is an RNAi inhibitor that targets Antithrombin mRNA. It is being studied in clinical trials for the treatment of Hemophilia A and Hemophilia B.
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Baseline phase 1 → approval rate
+9.6pp
Industry-wide phase 1 drugs reach approval ~9.6% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Big-pharma sponsor
+3.0pp
Sanofi 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 | 2033–2036 | — |
| EMA | EU | 2034–2037 | +0.7 yr |
| MHRA | GB | 2034–2037 | +0.7 yr |
| Health Canada | CA | 2034–2038 | +0.9 yr |
| TGA | AU | 2034–2038 | +1.2 yr |
| PMDA | JP | 2034–2038 | +1.5 yr |
| NMPA | CN | 2035–2039 | +2.3 yr |
| MFDS | KR | 2034–2038 | +1.4 yr |
| CDSCO | IN | 2034–2039 | +1.8 yr |
| ANVISA | BR | 2035–2039 | +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 | ALN-AT3SC |
|---|---|
| Sponsor | Sanofi |
| Drug class | RNA interference therapeutic |
| Modality | Small molecule |
| Phase | Phase 1 |
Mechanism of action
ALN-AT3SC is an RNAi therapeutic that targets messenger RNA encoding antithrombin, a natural anticoagulant protein. By reducing antithrombin levels, the drug aims to rebalance the coagulation system in patients with hemophilia and other bleeding disorders, potentially reducing bleeding episodes and the need for factor replacement therapy.
Approved indications
Common side effects
Key clinical trials
- A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors (PHASE3)
- A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors (PHASE3)
- A Phase 1 Study of an Investigational Drug, ALN-AT3SC, in Healthy Volunteers and Hemophilia A or B Patients (PHASE1)
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:
- ALN-AT3SC CI brief — competitive landscape report
- ALN-AT3SC updates RSS · CI watch RSS
- Sanofi portfolio CI
Frequently asked questions about ALN-AT3SC
What is ALN-AT3SC?
How does ALN-AT3SC work?
Who makes ALN-AT3SC?
What drug class is ALN-AT3SC in?
What development phase is ALN-AT3SC in?
Related
- Drug class: All RNA interference therapeutic drugs
- Manufacturer: Sanofi — full pipeline
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing