Horizon Scanning: FDA grants orphan drug status for ApoceptT to treat myelodysplastic syndrome
Source: BioSpace Area: News Apogenix GMbH, a clinical stage biopharmaceutical company, has announced that the FDA has granted orphan drug designation for ApoceptT for the treatment of myelodysplastic syndrome (MDS). A clinical phase I open-label trial is being initiated with ApoceptT in patients with MDS. Recruitment for the study began in January 2013. ApoceptT binds to the CD95 ligand (CD95L) and blocks the activation of the CD95 receptor. Excessive stimulation of the CD95 receptor on hematopoietic cells present in the bone marrow of MDS patients inhibits erythropoiesis. As a result, transfusion-dependent anemia develops, which is mostly refractory to erythropoiesis-stimulating agents.