Most patients with variant transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) who received tafamidis through an early access program maintained stable or improved heart failure symptoms, according to an abstract published in a supplement to the European Journal of Heart Failure.
Tafamidis, a transthyretin stabilizer, is approved for ATTR-CM and significantly reduced all-cause mortality at 30 months in the phase 3 ATTR-ACT trial. Trial completers were able to enter a long-term extension study, and a separate independent cohort gave patients who had not originally joined ATTR-ACT early access to the drug.
The entire independent early access cohort included 1476 patients with minimal entry criteria, but this analysis focused on a subset of 212 patients (14.4%) with variant ATTR-CM. Participants received open-label tafamidis free acid (61 mg once a day) for up to 60 months.