Dataset Information

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

ABSTRACT: 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.

SUBMITTER: Burton A

PROVIDER: S-EPMC7987260 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature

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Publications

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

Burton Arianna A Castaño Adam A Bruno Marianna M Riley Steve S Schumacher Jennifer J Sultan Marla B MB See Tai Sandi S Judge Daniel P DP Patel Jignesh K JK Kelly Jeffery W JW

Drug design, development and therapy 20210318

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). ...[more]

PMID: 33776421

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Dataset Information

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

Publications

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

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