Update: 30 May 2019
- Apotransferrinhas a physiological role in the transportation and distribution of iron among the body organs.
- Apotrasferrin has received orphan drug designation from EMA
- A proof of concept study in the Netherlands will begin in the second half of 2019 lasting 16 weeks with a maximum of 12 patients. Participants will receive apotrasferrin every two weeks to evaluate efficacy and safety in the treatment of patients with β-thalassaemia intermedia and analyse the impact of iron homeostasis and red blood cell function.
- The study expects to show that apotrasferrin treatment will reduce hemolysis and iron overload as well as extra-medullar hematopoiesis.