Tafamidis is recommended, within its marketing authorisation, as an option for treating wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR‑CM) in adults. Tafamidis is only recommended if the company provides it according to the commercial arrangement.
Why the committee made these recommendations
ATTR‑CM is a progressive condition that can lead to heart failure, but treatment options are limited to managing symptoms and best supportive care. Tafamidis is the first treatment for ATTR‑CM that aims to treat the condition.
Evidence from the main clinical trial shows that tafamidis reduces deaths and hospitalisations from conditions affecting the heart and blood vessels compared with placebo. But it is uncertain how long people live when having tafamidis and how long they take it for. Longer-term evidence from a different study reduces this uncertainty.
The most likely cost-effectiveness estimate for tafamidis is within the range that NICE considers an acceptable use of NHS resources. So, tafamidis is recommended.