Telik, Inc., a clinical stage drug development company, has received the US Food and Drug Administration (FDA) orphan drug designation for  its product candidate, ezatiostat HCL (Telintra), for the treatment of myelodysplastic syndrome (MDS).

Orphan designation grants potential US market exclusivity to a drug for the treatment of a specified condition for a period of seven years following FDA marketing approval.

Additional potential benefits of orphan designation include development grants, tax credits related to clinical trial expenses, protocol development assistance and exemption from FDA user fees. The US Orphan Drug Act aims to encourage the development of drugs for the diagnosis, prevention and treatment of medical conditions affecting fewer than 200,000 people in the US.

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.

Telik is a clinical stage drug development company focused on discovering and developing small molecule drugs to treat cancer. Telik’s product candidates were discovered using its proprietary drug discovery technology, TRAP, which enables the rapid and efficient discovery of small molecule drug candidates.