Telik制药宣布，FDA已授予Telintra(Ezatiostat HCl)孤儿药地位，用于骨髓增生异常综合症（myelodysplastic syndrome，MDS）的治疗。
Telintra是一种新颖的谷胱甘肽S-转移酶P1-1(glutathione S-transferase P1-1，GSTP1-1)抑制剂，能够可逆性抑制GSTP1-1，导致Jun激酶(JNK)的激活，该酶是血液前体细胞生长及分化的关键调节子。
英文原文：Palo Alto, CA – January 11, 2013 – Telik, Inc. (Nasdaq: TELK) was notified today that its product candidate, ezatiostat HCL (Telintra), has been granted orphan drug designation by the US Food and Drug Administration (FDA) 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.
