Margaret V. Ragni, MD, MPH

Ragni discussed unique advantages fitusiran offers over other approved therapies for hemophilia A or B.

Adding Treatment Options to the Hemophilia Landscape With Fitusiran’s Approval, with Margaret Ragni, MD, MPH

Last Friday, the FDA approved fitusiran, under the name Qfitlia, 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.

The FDA approved fitusiran’s biologics license application off data from the phase 3 ATLAS program. Recent data from the ATLAS-OLE trial (NCT03754790) were presented during December’s 

American Society of Hematology (ASH) Annual Meeting and Exposition

 and demonstrated that fitusiran prophylaxis, in combination with appropriate bleed management guidelines, allowed fewer infusions and lower doses of clotting factor concentrates (CFC) or bypassing agents (BPAs) to treat breakthrough bleeding in people with hemophilia A or B.

spoke with Margaret Ragni, MD, MPH, an investigator on the ATLAS program and Professor of Medicine and Clinical and Translational Research, Division Hematology/Oncology, University of Pittsburgh, and Director, Hemophilia Center of Western PA, to learn more about fitusiran and how the approval improves the treatment landscape for both hemophilia A and B.

The fitusiran regimen yielded a 70% reduction in mean annualized bleeding rate (ABR) compared with BPA prophylaxis (

 = .0002) and was comparable with CFC prophylaxis (

 = .61). People receiving the fitusiran regimen also had lower annualized mean weight-adjusted consumption of CFCs and BPAs.

“What it really does is it makes your life a little easier, because each time you have to treat it interrupts what you're doing in everyday life, and, especially if it's intravenous, that's a problem. So, [fitusiran is] a simplification of management of this disease, and you know, it's not for everybody. So, it's all about talking with your patient, understanding what their preferences are, and understanding whether they would tolerate this type of drug,” Ragni told 

Ragni's disclosures include Sanofi, Pfizer, Alnylam, CSL Behring, Novo Nordisk, and others.

FDA Approves Novel Treatment for Hemophilia A or B, with or without Factor Inhibitors. News release. FDA. March 28, 2025. https://www.fda.gov/news-events/press-announcements/fda-approves-novel-treatment-hemophilia-or-b-or-without-factor-inhibitors

Margaret V. Ragni, MD, MPH

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