Early Access to Tafamidis for Patients With Transthyretin Amyloid Cardiomyopathy.

ABSTRACT:

Background

Tafamidis is a standard of care treatment for patients with transthyretin amyloid cardiomyopathy (ATTR-CM). While evidence was being assessed by regulatory authorities, a new, independent, and inclusive cohort of the phase 3 long-term extension study offered early access to tafamidis.

Objectives

The purpose of this study was to present safety, mortality, and hospitalization findings for patients who received early access tafamidis.

Methods

Patients with ATTR-CM and who had not taken part in the phase 3 study were able to receive tafamidis free acid 61 mg (the dose later approved) for up to 60 months or until commercial availability in their region. Enrollment criteria were minimal.

Results

Among the 1,476 patients initiating tafamidis in the study be

SUBMITTER: Crespo-Leiro MG 

PROVIDER: S-EPMC12791842 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature