箭头制药公司收到ARO-AAT的孤儿药物名称

StephenW

February 15, 2018
Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-AAT
PASADENA, Calif.
(BUSINESS WIRE)
--Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced the
United States Food and Drug Administration (FDA) has granted orphan drug designation to ARO-AAT, Arrowhead's second-generation investigational medicine for the treatment of a rare genetic liver disease associated with alpha-1 antitrypsin
deficiency. Arrowhead filed a Clinical Trial Application in December 2017
requesting regulatory permission to begin first-in-human studies of ARO-AAT.
"The Orphan Drug Act provides important incentives for sponsors to develop drugs that treat rare diseases and we look forward to more engagement with the FDA as the development of ARO-AAT progresses," said Bruce D. Given, M.D., Arrowhead's chief operating officer and head of R&D.
The FDA Office of Orphan Products Development (OOPD) mission is to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task,
OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. Orphan drug designation
provides incentives for sponsors to develop products for rare diseases.

StephenW

2018年2月15日
箭头制药公司收到ARO-AAT的孤儿药物名称
帕萨迪纳，加利福尼亚州。
（美国商业资讯）
--Arrowhead制药公司（纳斯达克股票代码：ARWR）今天宣布
美国食品和药物管理局（FDA）已授予ARO-AAT的孤儿药物称号，ARO-AAT是Arrowhead的第二代研究药物，用于治疗与α-1抗胰蛋白酶相关的罕见遗传性肝病
不足。 Arrowhead于2017年12月提交临床试验申请
要求监管许可开始ARO-AAT的首次人体研究。
“孤儿药物法案为赞助商开发治疗罕见疾病的药物提供了重要的激励措施，并且随着ARO-AAT研发的进展，我们期待与FDA更多的合作，”Arrowhead首席运营官Bruce D. Given医学博士表示，研发主管。
FDA孤儿产品开发办公室（OOPD）的使命是推动评估和开发对罕见疾病或病症的诊断和/或治疗具有承诺的产品。为了完成这项任务，
OOPD对赞助商提交的科学和临床数据进行评估，以确定和指定对罕见疾病有前景的产品，并进一步推动这类有前途的医疗产品的科学发展。孤儿药指定
为赞助商开发针对罕见疾病开发产品的奖励。