Qfitlia

(Fitusiran)

Phase 3RNA therapy

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

Qfitlia is one of 16 sirna programs targeting Hemophilia. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

HemophiliaActive, not recruiting

HemophiliaPhase 3

Active, not recruiting

Competition

Competitive Landscape

16 competitors in Hemophilia

#DrugCompanyPhaseTrials

Videx

Bristol-Myers Squibb

Approved

1 2

Zerit

KKaruna Therapeutics, Inc., a Bristol Myers Squibb company

Approved

1 3

Hivid

CConrado Roberto Hoffmann Filho

Approved

1 4

Epivir

GGlaxo Dba Glaxosmithkline

Approved

1 5

Cyklokapron

JJ Molner

Approved

—6

Crixivan

CCidara Therapeutics Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)

Approved

1 7

Qfitlia

GGenzyme, a Sanofi Company

Phase 3

4 8

Antithrombin Concentrate (ATIIIC)

Sanofi

Phase 3

2 9

Foscenvivint

KKiminori Kimura, MD

Phase 2

1 10

Advil

KKenvue Brands

Preclinical

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

Timeline

Expected Milestones

Q1 2029

Phase 3Active, not recruiting

Hemophilia

NCT05662319

Q1 2030

ApprovedRecruiting

Hemophilia a

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 With Inhibitors

Completed· Sanofi logo

Sanofi·Phase 3·NCT03417102·60 enrolled

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

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

Completed· Sanofi logo

Sanofi·Phase 3·NCT03417245·120 enrolled

Show 1 more trial

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

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

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

An Open-label, 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

Completed· Sanofi logo

Sanofi·Phase 3·NCT03754790·281 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

Completed· Sanofi logo

Sanofi·Preclinical·NCT03974113·32 enrolled

Hemophilia A1 trial

An Open-label, Single-arm Treatment Study to Investigate the Safety and Tolerability of Switching From Emicizumab to Fitusiran Prophylaxis in Male Participants Aged ≥12 Years of Age With Severe Hemophilia A, With or Without Inhibitors

Recruiting· Sanofi logo

Sanofi·Approved·NCT06145373·20 enrolled

Regulatory

FDA Approval Timeline[4]

Mar 28, 2025

QFITLIA

NDA NDA219019

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]
PubMed — 10 publications indexed for Fitusiran. U.S. National Library of Medicine.
[7]
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

RE50996Mar 22, 2033

9127274Mar 22, 2033

11091759Dec 7, 2036

Key Publications[6]

METTL3-Mediated m6A Modification of COL5A2 Inhibits Ferroptosis Through an IGF2BP3-dependent Mechanism in Gastric Cancer.

Technol Cancer Res TreatDec 1, 2026DOI: 10.1177/15330338261481298

Clinical Severity and the Mental Health Treatment Gap Among Nursing Students: Findings From the Healthy Minds Study.

Nurse EducAug 28, 2026DOI: 10.1097/NNE.0000000000002304

The ongoing first-line treatment race for metastatic pancreatic ductal adenocarcinomas: will a tough disease lose its horror?

Future OncolAug 28, 2026DOI: 10.1080/14796694.2026.2725499

Advancing breast cancer drug delivery with polymeric layer-by-layer core-shell particles.

Expert Opin Drug DelivAug 28, 2026DOI: 10.1080/17425247.2026.2726476

Revisiting the role of danazol in the contemporary management of endometriosis-related pain: a systematic review.

Expert Opin PharmacotherAug 28, 2026DOI: 10.1080/14656566.2026.2726518

Potential drug-drug interactions and clinical outcomes among people living with HIV with comorbidities: A multicentre retrospective cohort study.

Br J Clin PharmacolAug 28, 2026DOI: 10.1002/bcp.70794

Pediatric developmental epileptic encephalopathies treated with cenobamate: Real-world outcomes across etiologies, syndromes, and seizure types.

EpilepsiaAug 28, 2026DOI: 10.1002/epi.70460

68Ga-FAPI PET/CT for assessing fibroblast activation and disease activity in primary Sjögren's disease: a prospective case-control study.

Rheumatology (Oxford)Aug 28, 2026DOI: 10.1093/rheumatology/keag440

Clinical and Epidemiological Characteristics of Infants <6 Months With Leukemia, Neuroblastoma, and Retinoblastoma in a Single Center in Iran.

J Pediatr Hematol OncolAug 28, 2026DOI: 10.1097/MPH.0000000000003268

Association of Imaging-Defined Deep Invasion and Tumor Volume With Clinical Cervical Lymph Node Metastasis in Hypopharyngeal Carcinoma.

J Comput Assist TomogrAug 28, 2026DOI: 10.1097/RCT.0000000000001919

Qfitlia

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q1 2029

Q1 2030

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]

Key Publications[6]

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