Addressing Unmet Needs in Hemophilia: Guy Young, MD


The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed the ATLAS-INH study of fitusiran.

“Bleeding can occur into the joints, which is a common location of bleeding, and that can lead to permanent joint damage. The ultimate goal is to prevent patients [with hemophilia A or B] from having any sort of permanent damage from any bleeds. To accomplish that goal, we need to minimize, if not eliminate, bleeds.”

People with hemophilia A or B with or without inhibitors treated with monthly prophylactic doses of fitusiran, an siRNA therapeutic targeting antithrombin, had reduced bleeding compared with those only given on-demand treatment, according to data from the phase 3 ATLAS-INH study (NCT03417102) presented at the 63rd Annual American Society of Hematology (ASH) Meeting, December 11-14, 2021.

The data were presented 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. Young and colleagues found that treated bleeding events were reduced to 0 in 25 treated patients (65.8%) and efficacy was seen in patients with both hemophilia A and hemophilia B. Treated participants also had higher physical health domain scores and health-related quality of life scores were also significantly higher (both P <.0001).

GeneTherapyLive spoke with Young to learn more about the ATLAS-INH study. He also discussed the goals of current clinical trials in hemophilia and unmet needs in the population.

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.
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