First Hemophilia A Gene Therapy Approved

The US Food and Drug Administration (FDA) approved Roctavian, a one-time gene therapy for adults with severe hemophilia A who do not have antibodies to the AAV5 virus. Roctavion uses a modified virus, called a vector, to deliver a working copy of the factor VIII gene to liver cells to enable the body to produc its own factor VIII.

The vector does not contain viral SNA and does not cause disease. Its safety and efficacy was evaluated in a multinational study in adult males aged 18 to 70 years with severe hemophilia A, who had previously been treated with factor VIII replacement therapy. After the infusion, the mean annual bleeding rate decreased from 5.4 to 2.6 bleeds per year. Most patients were given corticosteroids to suppress the immune system so that the gene therapy would be effective. For more info read here.

Source: LA Kelley Communications, Inc., Communique’