Fitusiran Approved for People With Hemophilia A or B, With and Without Inhibitors

The FDA has today 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.1

“Today’s approval of Qfitlia is significant for patients with hemophilia because it can be administered less frequently than other existing options,” Tanya Wroblewski, MD, deputy director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research, said in a statement.1 “This new treatment option highlights our continued efforts to improve the lives of patients with hemophilia.”

The FDA previously accepted Sanofi’s biologics license application (BLA) for fitusiran based in June 2024 based off data from the phase 3 ATLAS program, with a Prescription Drug User Fee Act (PDUFA) date of March 28.2 The latest, long-term findings from ATLAS were presented during December’s American Society of Hematology (ASH) Annual Meeting and Expositionby investigator Steven W. Pipe, MD, Professor and Laurence A. Boxer Research Professor of Pediatrics and Professor of Pathology, University of Michigan and CS Mott Children’s Hospital, Ann Arbor, Michigan.3

"It's another product that's available for our patients. We like to have lots of options for our patients... because there are certain situations where this may be better for some patients," Robert F. Sidonio Jr., MD, MSc, who is a Pediatric Hematologist/Oncologist, Medical Director of Hemophilia, and Medical Director of the Clinical Research Office at Aflac Cancer & Blood Disorders Center and Children's Healthcare of Atlanta, as well as an Associate Professor of Pediatrics at Emory University School of Medicine, told HCPLive. "Just telling a patient you only have to give maybe a total of 6 doses this year is pretty interesting when they obviously would have given a lot more than that before. So I think this is going to be a great option for some patients, and we're all happy to see this finally get to the finish line."

The data from the ATLAS-OLE trial (NCT03754790) 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.

Pipe and colleagues found that doses per bleed for participants without inhibitors and with inhibitors was reduced during fitusiran prophylaxis as compared to the CFC/BPA prophylaxis period, and overall, participants required fewer infusions for the management of breakthrough bleeds with fitusiran antithrombin-based dosing regimen (with inhibitors, 57; without inhibitors, 148) versus those who received CFC (n = 189) or BPA (n = 465) prophylaxis.3

The fitusiran regimen yielded a 70% reduction in mean annualized bleeding rate (ABR) compared with BPA prophylaxis (P = .0002) and was comparable with CFC prophylaxis (P = .61). People receiving the fitusiran regimen also had lower annualized mean weight-adjusted consumption of CFCs and BPAs.3

"[Fitusiran] adds another potential drug to our armamentarium that we have now," Margaret V. Ragni, MD, MPH, Professor of Medicine and Clinical and Translational Research, Division Hematology/Oncology, University of Pittsburgh, and Director, Hemophilia Center of Western PA, told HCPLive. "One of the interesting things, I think, is when some of the patients are not ready for gene therapy, it's nice to have these other agents that simplified treatment and yet didn't make them commit to something else that was not reversible, which is the gene therapy sort of approach."

REFERENCES

1. 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

2. Press Release: ISTH: Sanofi advances leadership in hemophilia with new data for ALTUVIIIO and fitusiran. News release. Sanofi. June 21, 2024. https://www.sanofi.com/en/media-room/press-releases/2024/2024-06-21-05-00-00-2902104

3. Pipe SW, Kavakli K, Matsushita T, et al. Reduced Doses of Factor Concentrates and Bypassing Agents to Treat Breakthrough Bleeds in Patients with Hemophilia A and B on Fitusiran Antithrombin-Based Dosing Regimen: ATLAS-OLE. Presented at: ASH Annual meeting; December 7-10; San Diego, California. Abstract 128.