Fitusiran in Hemophilia: A Novel Therapeutic Approach Targeting Antithrombin
4 affiliations
- 1From University of North Carolina Eshelman School of Pharmacy, Chapel Hill, North Carolina, and Carolinas Medical Center, Department of Pharmacy, Atrium Health of Advocate Health, Charlotte, North Carolina
- 2CHS Specialty Pharmacy Services, Atrium Health of Advocate Health, Charlotte, North Carolina
- 3Atrium Health Wake Forest Baptist of Advocate Health, Winston-Salem, North Carolina
- 4Atrium Health of Advocate Health, Charlotte, North Carolina
Abstract
Fitusiran was FDA approved on March 28, 2025, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors (neutralizing antibodies). This article contains an overview of hemophilia and current treatment and prophylactic strategies, highlighting the key differences between fitusiran and other agents used for prophylaxis. It summarizes antithrombin's role in the coagulation cascade and how fitusiran uses a novel mechanism as a small interfering RNA inhibitor that reduces expression of antithrombin. Pivotal clinical trials that led to its FDA approval are discussed, including dosing regimens, therapeutic drug monitoring, adverse effects, and clinical outcomes. This article provides in-depth information and clinical critique inclusive of and beyond the package insert guidance for advanced practitioners in hematology/oncology.
Related
- SAFETY AND EFFICACY OF THE FITUSIRAN ANTITHROMBIN-BASED DOSE REGIMEN IN PEOPLE WITH HEMOPHILIA A AND B BY AGE GROUP
- PO117 | LONGITUDINAL THROMBIN GENERATION PROFILING IN A PATIENT WITH SEVERE HEMOPHILIA A DURING ANTITHROMBIN-LOWERING THERAPY, CANCER, AND MAJOR SURGERY
- MAJOR ORTHOPEDIC SURGERIES CONDUCTED UNDER FITUSIRAN PROPHYLAXIS IN PEOPLE WITH HEMOPHILIA A AND B, WITH AND WITHOUT INHIBITORS
- PHASE 3 STUDY INVESTIGATING THE EFFICACY AND SAFETY OF FITUSIRAN PROPHYLAXIS IN MALE PARTICIPANTS AGED 1 TO <12 YEARS WITH HEMOPHILIA A/B, WITH OR WITHOUT INHIBITORS: ATLAS-KIDS, A TRIAL IN PROGRESS IN BRAZIL
- EXPERIENCE WITH MINOR SURGERIES IN PEOPLE WITH HEMOPHILIA A OR B WITH OR WITHOUT INHIBITORS RECEIVING FITUSIRAN
BibTeX
@article{dalton2026fitusiran,
title = {{Fitusiran in Hemophilia: A Novel Therapeutic Approach Targeting Antithrombin}},
author = {Anna Dalton and Ryan Clark and Justin Arnall and Christopher Parish and Lyndsi Roland},
journal = {Journal of the Advanced Practitioner in Oncology},
year = {2026},
month = sep,
doi = {10.6004/jadpro.2026.17.7.28},
url = {https://doi.org/10.6004/jadpro.2026.17.7.28},
note = {CC BY-NC-ND 3.0},
}
RIS
TY - JOUR
TI - Fitusiran in Hemophilia: A Novel Therapeutic Approach Targeting Antithrombin
AU - Anna Dalton
AU - Ryan Clark
AU - Justin Arnall
AU - Christopher Parish
AU - Lyndsi Roland
PY - 2026
DA - 2026/09/01
JO - Journal of the Advanced Practitioner in Oncology
DO - 10.6004/jadpro.2026.17.7.28
UR - https://doi.org/10.6004/jadpro.2026.17.7.28
AB - Fitusiran was FDA approved on March 28, 2025, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors (neutralizing antibodies). This article contains an overview of hemophilia and current treatment and prophylactic strategies, highlighting the key differences between fitusiran and other agents used for prophylaxis. It summarizes antithrombin's role in the coagulation cascade and how fitusiran uses a novel mechanism as a small interfering RNA inhibitor that reduces expression of antithrombin. Pivotal clinical trials that led to its FDA approval are discussed, including dosing regimens, therapeutic drug monitoring, adverse effects, and clinical outcomes. This article provides in-depth information and clinical critique inclusive of and beyond the package insert guidance for advanced practitioners in hematology/oncology.
SN - 2150-0878
SN - 2150-0886
ER -