The FDA approved the first gene therapy treatment for adults with severe hemophilia A.
Roctavian is an adeno-associated virus vector-based gene therapy that is administered as a single dose by intravenous infusion, according to a June 29 agency news release. It contains a viral vector carrying a gene for clotting, which is expressed in the liver to increase blood levels of factor VIII, a protein that enables blood clots, and reduces risk of uncontrolled bleeding.
In a trial of 112 patients with a three-year follow-up, the mean annual bleeding rate decreased from 5.4 bleeds per year to 2.6 bleeds.
Hemophilia A is a rare genetic bleeding disorder due to a mutation of the gene that produces factor VIII.