4.7 Review

Drug Discovery and Development in Rare Diseases: Taking a Closer Look at the Tafamidis Story

期刊

DRUG DESIGN DEVELOPMENT AND THERAPY
卷 15, 期 -, 页码 1225-1243

出版社

DOVE MEDICAL PRESS LTD
DOI: 10.2147/DDDT.S289772

关键词

amyloidosis; cardiomyopathy; heart failure; transthyretin; treatment; Vyndaqel/Vyndamax

向作者/读者索取更多资源

Rare diseases are considered a global public health priority, with Tafamidis representing a major breakthrough in the treatment of transthyretin amyloidosis after decades of research and clinical trials that established its safety and efficacy.
Rare diseases are increasingly recognized as a global public health priority. Governments worldwide currently provide important incentives to stimulate the discovery and development of orphan drugs for the treatment of these conditions, but substantial scientific, clinical, and regulatory challenges remain. Tafamidis is a first-in-class, disease-modifying transthyretin (TTR) kinetic stabilizer that represents a major breakthrough in the treatment of transthyretin amyloidosis (ATTR amyloidosis). ATTR amyloidosis is a rare, progressive, and fatal systemic disorder caused by aggregation of misfolded TTR and extracellular deposition of amyloid fibrils in various tissues and organs, including the heart and nervous systems. In this review, we present the successful development of tafamidis spanning 3 decades, marked by meticulous laboratory research into disease mechanisms and natural history, and innovative clinical study design and implementation. These efforts established the safety and efficacy profile of tafamidis, leading to its regulatory approval, and enabled post-approval initiatives that further support patients with ATTR amyloidosis.

作者

我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。

评论

主要评分

4.7
评分不足

次要评分

新颖性
-
重要性
-
科学严谨性
-
评价这篇论文

推荐

暂无数据
暂无数据