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FDA授予阿斯利康MEK抑制剂selumetinib孤儿药地位

新药编辑:新特药 更新时间:2015-04-18

2015年4月17日讯 /tumor.net.cn/ --英国制药巨头阿斯利康(AZN)近日接连收获FDA的孤儿药地位。昨日,肿瘤免疫疗法tremelimumab收获治疗恶性间皮瘤(malignant mesothelioma)的孤儿药地位。今日,口服小分子抑制剂selumetinib又收获了治疗葡萄膜黑色素瘤(uveal melanoma)的孤儿药地位。(相关阅读:FDA授予阿斯利康肿瘤免疫疗法tremelimumab孤儿药地位)

葡萄膜黑色素瘤是一种罕见、恶性程度极高的肿瘤,癌细胞在眼组织中形成,一旦扩散出眼组织,没有有效的治疗选择。该病是成人中最常见的原发性恶性眼内肿瘤,约占所有黑色素瘤病例的5%。

selumetinib由Array BioPharma发现,阿斯利康于2003年获得了该化合物的全球独家权利。selumetinib是一种口服、强效、选择性MEK抑制剂,能够抑制肿瘤细胞中的MEK通路,从而阻止肿瘤生长。MEK是MAPK通路的一部分,该通路在癌细胞中常常被激活,并且在许多不同实体瘤中升高,包括携带KRAS突变的肿瘤,该突变存在于20%的人类肿瘤,以及20%-30%的非小细胞肺癌(NSCLC)。

目前,阿斯利康正在开展一项III期临床研究,评估selumetinib联合化疗用于转移性葡萄膜黑色素瘤的一线治疗,该研究的数据预计在今年晚些时候获得。除了葡萄膜黑色素瘤,阿斯利康也正在III期临床调查selumetinib治疗KRAS突变阳性肺癌和甲状腺癌的治疗,同时也在一项II期研究中调查selumetinib用于I型神经纤维瘤病(neurofibromatosis,NF)儿科患者的治疗。

另外,评估selumetinib联合AZD9291(T790M定向EGFR抑制剂)及MEDI4736(抗PD-1)治疗非小细胞肺癌(NSCLC)的一项组合疗法研究的数据,将提交至2015年美国临床肿瘤学会(ASCO)年会。(tumor.net.cn)

英文原文:Selumetinib granted Orphan Drug Designation by US FDA for treatment of uveal melanoma

AstraZeneca today announced that the US Food and Drug Administration has granted Orphan Drug Designation for the MEK inhibitor selumetinib, for the treatment of uveal melanoma.

Uveal melanoma is a rare disease in which cancer cells form in the tissues of the eye. It is the most common primary intraocular malignancy in adults and comprises 5% of all melanomas1,2.

“Uveal melanoma is a rare and devastating disease for which there are currently no effective treatment options once it spreads beyond the tissues of the eye. Selumetinib could potentially become the first effective treatment for these patients. The Orphan Drug Designation is an important regulatory advancement as we further our development plans for selumetinib in uveal melanoma,” said Antoine Yver, Head of Oncology, Global Medicines Development at AstraZeneca.

The Orphan Drug Designation programme provides orphan status to drugs and biologics, which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US3.

Selumetinib, originally licensed from Array BioPharma Inc., inhibits the MEK pathway in cancer cells to prevent the tumour from growing. Data from a phase III study evaluating selumetinib in combination with chemotherapy in patients with first-line metastatic uveal melanoma is expected to be available later this year. In addition to uveal melanoma, selumetinib is being investigated in Phase III studies in KRAS mutation positive lung cancer and thyroid cancer and in Phase II in children with neurofibromatosis Type 1.

Initial data from a combination study of selumetinib with other AstraZeneca pipeline molecules including AZD9291 (T790M-directed EGFR inhibitor) and MEDI4736 (anti-PD-L1) in non-small cell lung cancer will be presented at the American Society of Clinical Oncology (ASCO) annual meeting 2015.

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