GREEN BAY, WI- Green Bay resident with Hemophilia A is the first patient in the state to receive gene therapy, Roctavian, since FDA approval.
“It is just exciting to know how far medicine has come. I never thought in my lifetime a treatment like this would become available. I have been through so many stages of advancements with hemophilia treatments over the course of my life and I’m so appreciative to be a recipient of the next stage with gene therapy. It gives me hope and hope for the Hemophilia community.”-David Novak, Patient of the Hemophilia Outreach Center in Green Bay with Hemophilia A.
“ROCTAVIAN represents a significant step forward in current treatment methods and a long-awaited breakthrough for the hemophilia A community. ,”stated Matthew Ryan, MD, Adult Hematologist and Medical Director at the Hemophilia Outreach Center in Green Bay, WI. “This innovative therapy provides patients with the possibility of living without frequent bleeding episodes or the inconvenience of regular infusions. The introduction of this one-time treatment also opens new discussions about treatment choices and goals in the community, which is crucial when managing a chronic condition, as individuals’ needs vary and evolve.”
“While there have been many recent advancements in the Hemophilia A space, it is exciting to see that gene therapy has finally arrived as this has been anticipated by both patients and providers for decades. This treatment option offers patients HOPE…the hope of decreasing and/or discontinuing prophylaxis and hope that they can reduce their annual bleed rates. What an improvement in a patient’s quality of life that would make!”- Andrea Buxton, APNP, Hemophilia Outreach Center.
The patient’s gene therapy infusion is scheduled for Thursday, May 9th at the Hemophilia Outreach Center (HOC) in Green Bay. HOC is a family-oriented organization committed to providing the highest quality comprehensive medical care, and advocacy for individuals with a bleeding disorder. HOC serves approximately 400 patients in Northeastern and Central Wisconsin. Learn more at: https://hemophiliaoutreach.org/
ROCTAVIAN (valoctocogene roxaparvovec-rvox), manufactured by BioMarin Pharmaceutical Inc., was FDA approved June of 2023 and is for the treatment of adults with severe hemophilia A who do not have antibodies to the virus, AAV5 which is determined by a blood test. Hemophilia A is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor VIII, a protein needed to produce blood clots to stop bleeding. Symptoms can include prolonged or heavy bleeding after an injury, surgery, or dental procedure; in severe cases, bleeding episodes can occur spontaneously without a clear cause. Prolonged bleeding episodes can lead to serious complications, such as bleeding into joints, muscles or internal organs, including the brain. Although ROCTAVIAN is not a cure for Hemophilia A patients, the medical advancement may reduce, if not, eliminate weekly preventative factor replacement and bleeds.
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