Drugs / Fitusiran
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed hemophilia B leyden 1 milestone | |||||
| Filed | US (FDA) | — | hemophilia B leyden | 2024-06-21 | “New Drug Application for fitusiran accepted for review by the US Food and Drug Administration” sanofi.com ↗ |
| Approved Indication not stated 1 milestone | |||||
| Approved | US (FDA) | QFITLIA | — | 2025-03-28 | fda.gov ↗ |
Trials 9
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT02554773 | Sep 2015 → Mar 2023 | hemophilia A, hemophilia B | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT03974113 | Jan 2020 → Aug 2026 | hemophilia | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 36 trials · 3 met primary | ||||||
| Phase 3 | NCT07285460 | Dec 2025 → Aug 2029 expected | hemophilia | Sanofi | Recruiting | No outcome recorded |
| Phase 3 | NCT05662319 | Feb 2023 → Mar 2027 expected | hemophilia | Sanofi | Active not recruiting | No outcome recorded |
| Phase 3 | NCT03754790 | Jan 2019 → Sep 2026 | hemophilia | Genzyme, a Sanofi Company | Completed | No outcome recorded |
| Phase 3 | NCT03549871 | Jul 2018 → Jan 2022 | hemophilia | Genzyme, a Sanofi Company | Completed | Met primary |
| Phase 3 | NCT03417245 | Mar 2018 → Jan 2021 | hemophilia A, hemophilia B | Genzyme, a Sanofi Company | Completed | Met primary |
| Phase 3 | NCT03417102 | Feb 2018 → Nov 2020 | hemophilia A, hemophilia B | Genzyme, a Sanofi Company | Completed | Met primary |
| Phase 41 trial | ||||||
| Phase 4 | NCT06145373 | Mar 2024 → May 2028 expected | hemophilia A | Sanofi | Recruiting | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 1 | NCT02035605 Comparator | Jan 2014 → Jul 2017 | hemophilia A, hemophilia B | Sanofi | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 8
| Date | Issuer | Release |
|---|---|---|
| 2025-12-11 | Sanofi | Regulatory Press Release: Sanofi’s Qfitlia and Cablivi approved in China, expanding care for rare diseases sanofi.com ↗
The National Medical Products Administration (NMPA) in China has approved two innovative Sanofi medicines for rare hematologic diseases: Qfitlia (fitusiran) for hemophilia and Cablivi (caplacizumab) for acquired thrombotic thrombocytopenic purpura. |
| 2025-03-28 | Sanofi | Regulatory Press Release: Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors sanofi.com ↗
Qfitlia approved as the first therapy in the US to treat hemophilia A or B with or without inhibitors |
| 2024-06-21 | Sanofi | Results Press Release: ISTH: Sanofi advances leadership in hemophilia with new data for ALTUVIIIO and fitusiran sanofi.com ↗
New ATLAS phase 3 study data reinforce the potential of fitusiran to provide prophylaxis for people with hemophilia A or B, with or without inhibitors |
| 2023-04-04 | Sanofi | Results Press Release: Two fitusiran Phase 3 studies published in The Lancet and The Lancet Haematology highlight potential to address unmet needs across all types of hemophilia sanofi.com ↗
Both Phase 3 studies achieved their primary and secondary endpoints; fitusiran prophylaxis demonstrated significant and clinically meaningful improvements in bleed protection across all hemophilia populations, presented at ASH 2021 |
| 2022-07-10 | Sanofi | Results Press Release: Fitusiran prophylaxis reduced bleeds by 61% in people with hemophilia A or B, with or without inhibitors, compared to prior factor or bypassing agent prophylaxis sanofi.com ↗
The study met the primary endpoint and demonstrated fitusiran prophylaxis significantly reduced bleeding episodes compared to prior factor or BPA prophylaxis. |
| 2021-12-14 | Sanofi | Results Data from two Phase 3 studies demonstrating fitusiran significantly reduced bleeds in people with hemophilia A or B, with or without inhibitors, were featured at ASH’s plenary and late-breaking sessions sanofi.com ↗
A >89% reduction in annualized bleeding rate was demonstrated with fitusiran prophylaxis in both studies when compared to the control arms |
| 2021-11-04 | Sanofi | Results New data in blood cancers, hemophilia, and other hematological disorders to be presented at ASH 2021 sanofi.com ↗ |
| 2020-06-19 | Sanofi | Results Sanofi announces positive long-term efficacy and safety data for fitusiran from interim analysis of Phase 2 extension study in people with hemophilia A and B, with or without inhibitors sanofi.com ↗
Long-term interim results from the Phase 2 open-label extension (OLE) study reinforce fitusiran’s potential to restore hemostatic balance and to lower annualized bleed rates (ABRs) over a period up to 57 months. |
All press releases naming this drug 10 releases
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 13 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | fitusiran | “Fitusiran, an investigational RNA interference (RNAi) therapy that targets antithrombin (encoded by SERPINC1), is in development to address these and other limitations.” PMID 28691885 ↗ Jul 2017 |
| Known as | ALN-57213 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297754 ↗ |
| Known as | ALN-AT3SC | “A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors” NCT03417245 ↗ |
| Known as | SAR439774 | “Fitusiran (SAR439774)” NCT02554773 ↗ |
| Known as | SAR439774, Qfitlia | “SAR439774, Qfitlia®” NCT06145373 ↗ |
| Action | Inhibit | “dose-dependent lowering of the antithrombin level” PMID 28691885 ↗ Jul 2017 |
| Modality | Small interfering RNA (siRNA) | “Fitusiran is an investigational small interfering RNA therapeutic that targets antithrombin” PMID 39642315 ↗ Mar 2025 |
| Route | Subcutaneous | “single subcutaneous injection of fitusiran” PMID 28691885 ↗ Jul 2017 |
| Target | SERPINC1 | “targets antithrombin (encoded by SERPINC1)” PMID 28691885 ↗ Jul 2017 |