Apitope International, a biotechnology company based in Diepenbeek, Belgium has received Orphan Drug Designation from the U.S. FDA for ATX-F8-117 for the treatment of Hemophilia A.  Hemophilia A is a rare genetic and chronic bleeding disorder caused by mutation in the gene encoding coagulation factor VIII.  Depending on the nature of the mutations in the gene and the consequent residual activity of factor VIII in the plasma, the severity of the clinical manifestation can be quite heterogeneous.  Hemophilia A patients are normally managed by infusion of coagulation factor VIII to alleviate clotting problems associated with factor VIII deficiency.  However, some patients develop inhibitor antibodies against wild type factor VIII over time and this complication significantly limits the treatment of this disorder.  ATX-F8-117 is composed of peptides derived from factor VIII and has the potential to treat hemophilia A patients and simultaneously prevent inhibitor antibody development.  Currently, there are few effective therapies available to treat patients who have developed factor VIII infusion-related complications making ATX-F8-117 potentially life changing therapy for these patients.

 

References:

  1. http://apitope.com/apitope-receives-orphan-designation-european-union-atx-f8-117-treatment-haemophilia/
  2. http://omim.org/entry/306700