Green Bay Resident to Become First Wisconsinite to Receive Gene Therapy Treatment for Hemophilia A
A Green Bay resident will become the first Wisconsinite to receive a gene therapy treatment for hemophilia A since it was approved by the FDA in June.
David Novak, a patient at the Hemophilia Outreach Center in Green Bay, will receive the gene therapy infusion, called ROCTAVIAN, on Thursday, May 9th.
“It is just exciting to know how far medicine has come,” said Novak, who has Hemophilia A. “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.”
Hemophilia A is a genetic bleeding disorder that results from missing/insufficient levels of the blood clotting Factor VII. This is a protein that is needed to produce the blood clots that stop bleeding. The most common symptom is prolonged or heavy bleeding following an injury or medical procedure, but in severe cases, a bleeding episode can occur without any cause. These prolonged episodes can lead to blood getting into joints, organs, muscles, and even the brain.
“ROCTAVIAN represents a significant step forward in current treatment methods and a long-awaited breakthrough for the hemophilia A community,” said Matthew Ryan, MD, an adult hematologist and the medical director at the Hemophilia Outreach Center. “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,” said Andrea Buxton, an advanced practice nurse prescriber at the Hemophilia Outreach Center. “This treatment option offers patients HOPE…the hope of decreasing and/or discontinuing prophylaxis and hope that they can reduce their annual bleed rates.”
ROCTAVIAN (valoctocogene roxaparvovec) was manufactured by BioMarin Pharmaceutical Inc. and FDA-approved in June of 2023. It’s intended for the treatment of adults with severe hemophilia. It is not a cure for the disorder, but may eliminate or reduce the need for weekly preventative treatment of hemophilia A.
Source: WBAY-TV 2 Green Bay (ABC Affiliate)