葛兰素史克（GSK）2月28日宣布，已向FDA提交了药物Promacta（eltrombopag，艾曲波帕）的补充新药申请（sNDA），寻求批准该药用于对免疫抑制疗法（IST）响应不足的重型再生障碍性贫血（SAA）患者血细胞减少症（cytopenia）的治疗。

此前，FDA已于今年2月授予Promacta治疗SAA的突破性疗法认定。Promacta sNDA的提交，是基于一项开放标签II期NIH研究的数据，该项研究在43例对IST响应不足的SAA患者中开展。

重型再生障碍性贫血（SAA）是一种罕见性疾病，患者骨髓无法制造足够的新的血细胞。目前，还没有药物获批用于对免疫抑制疗法（IST）无响应的SAA患者的治疗。对初始IST响应不足的SAA患者群体，约40%的患者会在疾病确诊5年内，死于感染或出血。

除了通过突破性治疗药物资格而获得加速审评之外，葛兰素史克还能在审评过程中期待更多来自FDA的指导。

如果获得批准，这将是艾曲波帕在美国第三次获得推荐，之前这款药物获批用于治疗患有慢性丙型肝炎（用干扰素之前或期间）或慢性免疫血小板减少症成人患者的血细胞计数减少，适用于对某些治疗药物不再起作用的患者。

葛兰素史克与Ligand公司合作开发了这款药物，Ligand将收取艾曲波帕的销售提成。美国之外市场，这款药物被称作Revolade。

关于

目前，Eltrombopag已获全球100多个国家批准，用于慢性免疫（特发性）血小板减少性紫癜（ITP）患者血小板减少症（thrombocytopenia）的治疗，同时已获43个国家批准用于慢性丙型肝炎（CHC）患者血小板减少症的治疗，以便启动并维持以干扰素为基础的肝病标准疗法。

该药在美国的商品名为Promacta，在欧洲及其他国家和地区的商品名为Revolade。

GSK announces submission to U.S. regulatory authorities for Promacta(eltrombopag) for severe aplastic anaemia

Issued: 28 February 2014, London UK

GlaxoSmithKline plc (LSE:GSK) announced today the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for Promacta? (eltrombopag) for the treatment of cytopenias (a reduction in blood cells) in patients with severe aplastic anaemia (SAA) who have had an insufficient response to immunosuppressive therapy (IST).

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 IST. Of those patients unresponsive to initial IST, approximately 40% die from infection or bleeding within 5 years of their diagnosis.[i]

On 3 February 2014, GSK announced that the FDA granted Breakthrough Therapy designation for Promacta in SAA.

The sNDA application is based on the results from an open-label, Phase II National Institute of Health (NIH) study of eltrombopag in 43 heavily pre-treated SAA patients with an insufficient response to IST.

About Eltrombopag

Eltrombopag—marketed as Promacta in the U.S. and as Revolade? in Europe and other countries across the world—is not approved or licensed anywhere in the world for use in severe aplastic anaemia.

Eltrombopag is approved in 100 countries worldwide for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) and in 43 countries worldwide for the treatment of for the treatment of thrombocytopenia (low blood platelet counts) in patients with chronic hepatitis C to allow them to initiate and maintain interferon-based therapy.

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