Researchers discovered a clinically translatable gene therapy protocol, which is a gene delivery system that introduced the production of therapeutic clotting proteins in platelets, so that the inhibition of inactivating antibodies is avoided.
Over the past few months, the Food and Drug Administration (FDA) approved several drugs for the control and prevention of bleeding episodes in patients with hemophilia. Other drug classifications granted included Orphan Drug Status and Breakthrough Therapy designation.
Seven patients have been treated successfully for hemophilia B and so far essentially none have developed inhibitors, according to the results of phase I and II trials of Baxalta drug BAX 335.
An analysis of randomized controlled trials (RCTs) published by the Institute for Quality and Efficiency in Health Care (IQWiG) determined that prophylactic treatment for hemophilia A is superior to on demand treatment in some outcomes.