Telik, a clinical stage drug development company focused on developing small molecule drugs to treat cancer, was notified today that its product candidate, ezatiostat HCL (Telintra), has been granted Orphan Drug designation by the FDA for the treatment of myelodysplastic syndrome (MDS).
Telintra is an investigational agent in development for the treatment of MDS and idiopathic chronic neutropenia. Telintra is a novel inhibitor of the enzyme glutathione S-transferase P1-1, leading to activation of Jun kinase, a key regulator of cellular growth and differentiation of blood precursor cells. Telintra has been shown to cause clinically significant and sustained reduction in red blood cell transfusions, transfusion independence and multilineage responses in MDS patients. The results of four clinical trials of Telintra in MDS have been reported in peer-reviewed scientific journals.
Orphan designation grants potential U.S. market exclusivity to a drug for the treatment of a specified condition for a period of seven years following FDA marketing approval.