RIYADH — Saudi Food and Drug Authority (SFDA) has recently approved two drugs for the treatment and prophylaxis of people with tendency to bleed, i.e., hemophilia A and B. A medical meeting held in Riyadh recently among experts in hematology in Saudi Arabia and the UK in conjunction with the 11th Congress of the Saudi Society of Hematology, noted that the new treatments are recombinants DNA-derived factor VIII and factor IX to replace missing clotting factors in hemophilic patients. They are developed through a state-of-the-art manufacturing and purification process that helps reduce the risk of serious viral infections that might be transmitted via infected blood. “Hemophilia treatments have witnessed exceptional developments following the introduction of plasma-derived options. However, there has been always a need for effective treatments with high safety and efficacy profile that could minimize risk of serious viral infections including HBV, HCV and HIV etc.” said Dr. Tarek Owaidah, Consultant Hematologist, Pathology Lab. Medicine, King Faisal Specialist Hospital and Research Centre. “Scientists then developed recombinants derived from human or animal sources. When selecting a plasma-derived or recombinant concentrates, the two most important safety issues are transmission of infectious agents and inhibitor formation.” Hemophilia is an inherited disease that prevents the blood from clotting properly. People with hemophilia have deficiency blood protein also called clotting factor which is necessary to clot the blood and stop bleeding. Most patients with hemophilia A are dependent on factor VIII replacement therapy, while patients with hemophilia B are dependent on factor IX replacement therapy. According to the World Federation of Hemophilia, 80 percent of hemophilic patients are afflicted with hemophilia A while 20 percent of those patients have hemophilia B. Recent studies showed that approximately 320,000 – 340,000 persons – predominantly males – are affected with hemophilia A worldwide. In Saudi Arabia, with a clear absence of accurate statistics of patients with hemophilia however, sporadic studies reveal that the number of those patients may go up to 2000. There are quite a few elements to be considered in plasma-derived treatments including country of plasma origin, donor selection, viral screening by antibody, direct viral testing and viral screening and inactivation in pooled. Similarly, there are a few considerations for recombinant treatments including eliminating exogenous human or animal proteins and viral removal and inactivation steps reducing. The newly approved completely albumin-free treatments represent the latest generation of recombinants that employ advancements of manufacturing and purification processes. — SG
