FDA授予GSK eltrombopag突破性疗法认定

作者：tomato来源：生物谷2014-2-12

                               
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2014年2月11日讯 /生物谷BIOON/ --葛兰素史克（GSK）2月3日宣布，FDA已授予药物Promacta/Revolade（eltrombopag，艾曲波帕）突破性疗法认定，用于对免疫抑制疗法反应不足的重症再生障碍性贫血（SAA）患者血细胞减少症（cytopenias）的治疗。

eltrombopag突破性疗法的授予，是基于在43例对初始IST治疗无响应的SAA患者中开展的II期NIH研究（09-H-0514）的数据。

重症再生障碍性贫血（SAA）是一种罕见疾病，患者骨髓无法产生足够的新血细胞。目前，尚未有药物获批用于对初始免疫抑制疗法（IST）无响应的SAA患者的治疗，这类患者在确诊后，约有40%的患者在5年内会死于感染或出血。

目前，eltrombopag已获FDA批准，以商品名Promacta上市，同时获欧盟批准，以商品名Revolade上市，用于慢性免疫（特发性）血小板减少症（ITP）的治疗。此外，eltrombopag于2013年9月获欧盟批准，用于慢性丙型肝炎（HCV）成人患者血小板减少症（thrombocytopenia）的治疗。

英文原文：GSK gains FDA Breakthrough Therapy designation for Promacta®/Revolade® (eltrombopag) for severe aplastic anaemia

GlaxoSmithKline plc (LSE:GSK) announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for Promacta®/Revolade® (eltrombopag) for the treatment of cytopenias in patients with severe aplastic anaemia (SAA) who have had insufficient response to immunosuppressive therapy.  Eltrombopag is not approved or licensed anywhere in the world for use in this treatment setting. Breakthrough Therapy Designation is the newest of the FDA’s programmes aimed at accelerating the development and review times of drugs for serious or life-threatening conditions.

SAA is a rare disorder in which the bone marrow fails to make enough new blood cells. There are no approved therapies available for SAA patients unresponsive to initial immunosuppressive therapy (IST). Of those patients unresponsive to initial IST, approximately 40% die from infection or bleeding within 5 years of their diagnosis.

The Breakthrough Therapy designation was based on the results from an open-label, Phase II National Institute of Health (NIH) study (09-H-0154) of eltrombopag in 43 heavily pre-treated SAA patients with an insufficient response to IST.

(责任编辑：lishuheng)