Clinical trial Apotransferrin treatment in patients with β-thalassemia

Amsterdam – April 26, 2019 – Sanquin Plasma Products announced the first patient enrollment in a Proof of Concept clinical trial. The clinical trial evaluates the efficacy and safety of Apotransferrin treatment in patients with β(beta)-thalassemia intermedia. In total twelve patients of a single Dutch University Medical Center will be enrolled.

β-thalassemia is an inherited and life-long debilitating disorder characterized by a deficiency of hemoglobin synthesis, which carries and supplies oxygen throughout the human body. The total annual incidence of symptomatic cases is estimated at 1 in 100,000 worldwide and 1 in 10,000 in the European Union.  

“Thalassemia is one of the most prevalent genetic forms of anemia in the world and is complicated by severe morbidity and early mortality related to chronic anemia and iron overload. In the current study, we expect to demonstrate that Apotransferrin treatment will not only reduce hemolysis in both transfusion-dependent as well as transfusion-independent thalassemia but also reduce iron overload and extra-medullar hematopoiesis. Apotransferrin is therefore expected to have a disease modifying effect on this inherited disease”, said B.J. Biemond, MD, PhD, Professor of Hematology at the Amsterdam University Medical Center and principal investigator of the clinical trial. 

Sanquin Plasma Products manufactures and distributes medicinal products derived from human plasma and indicated for a variety of diseases. The European Commission, acting on the positive recommendation from the European Medicines Agency (EMA), has granted an orphan drug designation for Apotransferrin, made by Sanquin Plasma Products, to treat rare conditions such as β-thalassemia and hypotransferrinemia. 

“Sanquin Plasma Products has developed a state-of-the-art method to extract and purify pharmaceutical grade Apotransferrin from human plasma. Thanks to continuous research into further application of this protein, Apotransferrin has the potential to improve the treatment of severe rare diseases”, said T. Mulders, PhD, Unit Director of Medical and Regulatory Affairs at Sanquin Plasma Products.