Qfitlia

(Fitusiran)

Phase 3RNA therapy

Qfitlia is a rna therapy that works as Antithrombin Sirna.[2] It is being investigated across 3 indications, with its most advanced program in Phase 3.[1]

Qfitlia is one of 75 sirna programs targeting Hemophilia. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

HemophiliaActive, not recruiting

Hemophilia aActive, not recruiting

Severe Hemophilia aActive, not recruiting

HemophiliaPhase 3

Active, not recruiting

Hemophilia aPhase 3

Active, not recruiting

Severe Hemophilia aPhase 3

Active, not recruiting

Competition

Competitive Landscape

93 competitors in Hemophilia a

#DrugCompanyPhaseTrials

Hemlibra

Roche

Phase 3

16 2

Recombinant Human Coagulation FVIII

Chia Tai Tianqing Pharmaceutical Group Co., Ltd.

Phase 3

2 3

Eptacog Alfa

Novo Nordisk

Phase 3

1 4

Qfitlia

GGenzyme, a Sanofi Company

Phase 3

1 5

Valoctocogene Roxaparvovec

BioMarin Pharmaceutical

Phase 3

3 6

Recombinant Factor VIII

Bayer

Phase 3

2 7

Translarna

PPTC Therapeutics

Phase 2

1 8

Rfviia

Roche

Phase 3

1 9

Marstacimab

Pfizer

Phase 3

1 10

PF-06741086

Pfizer

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 3Active, not recruiting

Hemophilia a

NCT03754790

PreclinicalActive, not recruiting

Hemophilia

NCT03974113

Q1 2029

Phase 3Active, not recruiting

Hemophilia

NCT05662319

Q4 2029

Post-ApprovalRecruiting

Hemophilia

NCT06145373

Q4 2031

Phase 3Recruiting

Hemophilia

NCT07285460

Clinical

Trials by Indication[1]

Severe Hemophilia a4 trials

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

Completed· Sanofi logo

Sanofi·Phase 3·NCT03417245·120 enrolled

A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis

Completed· Sanofi logo

Sanofi·Phase 3·NCT03549871·80 enrolled

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

Completed· Sanofi logo

Sanofi·Phase 3·NCT03417102·60 enrolled

Show 1 more trial

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

Completed· Sanofi logo

Sanofi·Phase 1/2·NCT02554773·34 enrolled

Hemophilia4 trials

A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia

Active, not recruiting· Sanofi logo

Sanofi·Phase 3·NCT05662319·91 enrolled

A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab

Recruiting· Sanofi logo

Sanofi·Phase 4·NCT06145373·20 enrolled

An Open-label, Parallel, Phase 3, Two-arm Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B With or Without Inhibitory Antibodies to Factors VIII or IX

Recruiting· Sanofi logo

Sanofi·Phase 3·NCT07285460·85 enrolled

Show 1 more trial

ATLAS-PEDS: An Open-label, Multinational Study of Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

Active, not recruiting· Sanofi logo

Sanofi·Preclinical·NCT03974113·32 enrolled

Hemophilia A1 trial

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

Active, not recruiting· Sanofi logo

Sanofi·Phase 3·NCT03754790·281 enrolled

Regulatory

FDA Approval Timeline[4]

Mar 28, 2025

QFITLIA

NDA NDA219019

Sources

References

[1]
ClinicalTrials.gov — 9 trial records for Fitusiran. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Fitusiran compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Fitusiran. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA219019). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Qfitlia. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Genzyme, a Sanofi Company company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Antithrombin Sirna[2]
Modality: RNA therapy[2]
Highest Phase: Phase 3[1]
Company: GGenzyme, a Sanofi Company

Patent Landscape[5]

Patent Cliff

Mar 15, 2033

Earliest patent expiration — generic entry possible after this date

Patent #Expires

9376680Mar 15, 2033

9127274Mar 22, 2033

11091759Dec 7, 2036

Qfitlia

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q1 2029

Q4 2029

Q4 2031

Trials by Indication[1]

Severe Hemophilia a4 trials

Hemophilia4 trials

Hemophilia A1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

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