SFDA approves two hemophilia drugs

The Saudi Food and Drug Authority (SFDA) has approved two drugs used for the treatment and prophylaxis of people with blood clotting disorder, i.e., hemophilia A and B. This was disclosed at a medical conference in Riyadh Tuesday.
The meeting was attended by experts in hematology in the Kingdom and the United Kingdom in conjunction with the 11th Congress of the Saudi Society of Hematology that was held in Riyadh on Wednesday.
Dr. Charles R.M. Hay from the Central Manchester University Hospitals was present to speak about hemophilia A and B.
Hemophilia is an inherited disease that prevents the blood from clotting properly. People with hemophilia have a deficiency in blood protein, also called clotting factor, which is necessary to stop bleeding and clot the blood. 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 have hemophilia B. Recent studies showed that approximately 320,000 to 340,000 persons — predominantly males — are affected with hemophilia A worldwide.
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.
Speaking to Arab News, Hay said that in the Kingdom, with a population of 29 million, there should be at least 3,000 patients suffering from hemophilia. “I presume half of them must be severely affected,” Hay said, indicating that the percentage of patients is the same in all countries.
“Hemophilia treatments have witnessed exceptional developments following the introduction of plasma-derived options. However, there has always been need for effective treatments with a high safety and efficacy profile that could minimize risk of serious viral infections including HBV, HCV and HIV,” Dr. Tarek Owaidah, consultant hematologist at King Faisal Specialist Hospital & Research Center, said yesterday.
“Scientists then developed recombinants derived from human or animal sources. When selecting a plasma-derived or recombinant concentrate, the two most important safety issues are transmission of infectious agents and inhibitor formation.”
The newly approved treatments represent the latest generation of recombinants that employ advancements of manufacturing and purification processes. Using a synthetic ligand that is free of animal materials and has gone through multiple purification steps including chromatography and nanofiltration, these treatments help to further reduce the risk of potential viral contamination. They are the only recombinant treatments that go through a nanofiltration purification step, which is an additional virus removal stage that includes non-enveloped viruses and possible new infectious agents.
According to Owaidah, a few decades ago, children with hemophilia had significantly reduced life expectancy and suffered from arthritis and joint deformity. However, with the introduction of new biotechnologically advanced treatment options, hemophilic patients became more active members in society with increased life expectancy.
“When it comes to treatment, safety is a primary concern for patients. We believe that the state-of-the-art manufacturing and purification process used for the new recombinants make them an effective and safe therapeutic option for many patients with hemophilia,” concluded Owaidah.