GSHTC debuts novel hemophilia gene therapy
A novel hemophilia gene therapy drug has been successfully administered to the first patient in Texas outside of a clinical trial setting by physicians at McGovern Medical School and the Gulf States Hemophilia and Thrombophilia Center.
The patient was administered the FDA-approved infusion, Roctavian™, to treat hemophilia A. The drug was approved by the United States Food and Drug Administration in June 2023.
Hemophilia is a blood disorder that can be passed from a patient to their child through genetics. It is caused by a mutation or change in one of the genes that prevents the clotting protein from working properly or being missing altogether, which then may lead to spontaneous bleeding. If not diagnosed and treated early, hemophilia can lead to crippling pain, internal bleeding into muscles and joints, severe joint damage, disability, and even early death.
Hemophilia A, the most common of the two types of hemophilia, is estimated to affect as many as 33,000 males in the United States, according to the Centers for Disease Control and Prevention. The novel gene therapy addresses the underlying genetic cause of Hemophilia A and has the ability to reduce the need for traditional clotting factor replacement therapies.
“The treatment for the patient marked a significant milestone for this program. The patient was incredibly appreciative of walking through this journey with us,” said Miguel Escobar, MD, professor in the Department of Pediatrics and medical director of the Gulf States Hemophilia and Thrombophilia Center.
“After many months of discussion and preparation, this would not have been possible without the exceptional teamwork displayed by all of our staff. Being able to prepare the drug in our university pharmacy and then infuse it within the clinic represents the dedication of this team.”