Statement: HFA Responds to Latest Gene Therapy Announcement

Yesterday, the U.S. Food and Drug Administration approved Roctavian (valoctocogene roxaparvovec), the first gene therapy for hemophilia A. This new treatment option, for the treatment of adults with severe factor VIII deficiency, represents a significant milestone in the treatment of hemophilia A.   

As a community-based, patient-centered organization, Hemophilia Federation of America (HFA) intimately understands and recognizes hemophilia’s profound impact on the physical, emotional, social, and financial well-being of affected individuals and families. HFA shares our members’ longstanding hope for a safe, effective, single-dose therapy that could eliminate bleeding, improve quality of life, and allow those affected by hemophilia to thrive. 

HFA welcomes innovations in hemophilia treatment, and at the same time recognizes that uncertainties will persist with respect to new therapies: durability, variability, eligibility for treatment and more. Patients and providers should thoughtfully weigh whether gene therapy is appropriate for their individual situations and treatment goals. In all cases, the decision whether or not to opt for gene therapy must rest with the patient and their health care provider. 

As HFA acknowledges this potentially promising advancement in hemophilia treatment, we remain mindful of our community’s history. We will continue our work to ensure that patient health, safety, and interests always come first. 

Source: Hemophilia Federation of America, June 2023