Vutrisiran: First Approval

被引:94
作者
Keam, Susan J. [1 ]
机构
[1] Springer Nat, Private Bag 65901, Auckland 0754, New Zealand
关键词
AMYLOIDOSIS;
D O I
10.1007/s40265-022-01765-5
中图分类号
R9 [药学];
学科分类号
1007 ;
摘要
Vutrisiran (AMVUTTRA (TM)) is a subcutaneously administered transthyretin-directed small interfering ribonucleic acid (siRNA) therapeutic (also called RNA interference, or RNAi therapeutic) being developed by Alnylam Pharmaceuticals, Inc. for the treatment of amyloid transthyretin-mediated (ATTR) amyloidosis, including hereditary ATTR (hATTR) amyloidosis and wild-type ATTR (wtATTR) amyloidosis. Vutrisiran was approved in June 2022 in the USA for the treatment of the polyneuropathy of hATTR amyloidosis in adults and received a positive opinion in the EU in July 2022 for the treatment of hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. Vutrisiran is also under regulatory review for the treatment of the polyneuropathy of hATTR amyloidosis in adults in Japan and Brazil. This article summarizes the milestones in the development of vutrisiran leading to this first approval in hATTR amyloidosis.
引用
收藏
页码:1419 / 1425
页数:7
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