Drugs / Fitusiran
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Fitusiran

Small interfering RNA (siRNA) targets SERPINC1 via inhibition

Developed for
hemophilia · hemophilia A · hemophilia B
Investigated by
Genzyme, a Sanofi Company · Sanofi

Regulatory milestones approvals, filings & regulatory actions · 1 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved US (FDA) QFITLIA 2025-03-28 fda.gov

Trials 9 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
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 overdue hemophilia Genzyme, a Sanofi Company Active not recruiting No outcome recorded
Phase 36 trials
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 → Nov 2026 expected hemophilia Genzyme, a Sanofi Company Active not recruiting No outcome recorded
Phase 3 NCT03549871 Jul 2018 → Jan 2022 hemophilia Genzyme, a Sanofi Company Completed No outcome recorded
Phase 3 NCT03417245 Mar 2018 → Jan 2021 hemophilia A, hemophilia B Genzyme, a Sanofi Company Completed No outcome recorded
Phase 3 NCT03417102 Feb 2018 → Nov 2020 hemophilia A, hemophilia B Genzyme, a Sanofi Company Completed No outcome recorded
Phase 41 trial
Phase 4 NCT06145373 Mar 2024 → May 2028 expected hemophilia A Sanofi Recruiting No outcome recorded
Also used as a comparator or background therapy in 1 trial
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 1 NCT02035605 Comparator Jan 2014 → Jul 2017 hemophilia A, hemophilia B Sanofi Completed No outcome recorded

Press releases naming this drug 7 releases

DateIssuerRelease
2024-06-21 Sanofi Press Release: ISTH: Sanofi advances leadership in hemophilia with new data for ALTUVIIIO and fitusiran sanofi.com
2023-04-04 Sanofi 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
2022-07-10 Sanofi 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
2021-12-14 Sanofi 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
2021-02-05 Sanofi Sanofi presents amended protocols in fitusiran clinical studies at EAHAD 2021 sanofi.com
2020-12-10 Sanofi Sanofi to resume dosing in fitusiran clinical studies in the U.S. sanofi.com
2020-06-19 Sanofi 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

Evidence & citations 9 cited values

Every value below carries the sentence it was read from. 13 sources stand behind the page.

FieldValueCited 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
9

NCT07285460

NCT02554773

NCT03417102

NCT03754790

NCT03417245

NCT03974113

NCT03549871

NCT06145373

NCT05662319

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
1

NCT02035605

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