Improving Outcomes in Hemophilia With Fitusiran: Guy Young, MD

Video

The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed advantages of the siRNA therapeutic.

“We had a decent number of patients with hemophilia B with inhibitors in the study. That is a very small population in the United States, roughly 150 patients. These patients have no good alternatives to preventing bleeding and few alternatives to treating bleeding. That's the group of patients that continues to really suffer and has been left behind... by the innovations we've had with emicizumab and factor therapy in hemophilia A.”

Findings from the phase 3 ATLAS-INH study (NCT03417102) of fitusiran were presented at the 63rd Annual American Society of Hematology (ASH) Meeting, December 11-14, 2021, by Guy Young, MD, director, Hemostasis and Thrombosis Program, Children’s Hospital of Los Angeles and professor of Pediatrics, Keck School of Medicine, University of Southern California. Fitusiran reduced bleeding in people with hemophilia A or B with or without inhibitors compared with those only given on-demand treatment. The siRNA therapeutic targets antithrombin and is administered in monthly, prophylactic doses.

Most participants in the study (65.8%) with both hemophilia A and B had treated bleeding events reduced to 0. In general, treated participants also had higher physical health domain health-related quality of life scores (both P <.0001).

GeneTherapyLive spoke with Young to learn more about fitusiran and how the siRNA therapeutic may improve outcomes in hemophilia A and B. He discussed unmet needs in hemophilia B and the decent population included in the ATLAS-INH study.

REFERENCE
Young G, Srivastava A, Kavkli K, et al. Efficacy and safety of fitusiran prophylaxis, an siRNA therapeutic, in a multicenter phase 3 study (ATLAS-INH) in people with hemophilia A or B, with inhibitors (PwHI). Presented at: 63rd Annual ASH Meeting; December 11-14, 2021, Atlanta, GA. Abstract 4.

Newsletter

Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.

Recent Videos
Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira, and Kilian Guse, PhD, the vice president of genetic medicine platforms at Pacira
Derek Jackson, BS, MA, the vice president of cell & gene therapy product development at Pacira
Jeffrey Chamberlain, PhD
Tami John, MD
Tami John, MD
Tami John, MD
Matthew Ku, MBBS, FRACP, RACP, FRCPA/RCPA, PhD, an associate professor and the lymphoma stream lead at St Vincent’s Hospital
Saurabh Dahiya, MD, FACP, an associate professor of medicine at Stanford University School of Medicine; as well as clinical director of Cancer Cell Therapy in the Division of Blood and Marrow Transplantation and Cell Therapy at Stanford Medicine
Shahzad Raza, MD, a hematologist/oncologist at the Cleveland Clinic
Manali Kamdar, MD, the associate professor of medicine–hematology and clinical director of lymphoma services at the University of Colorado
Related Content
© 2025 MJH Life Sciences

All rights reserved.