Tafamidis for treating transthyretin amyloidosis with cardiomyopathy

1.1 Tafamidis is not recommended, within its marketing authorisation, for treating wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR‑CM) in adults.

1.2 This recommendation is not intended to affect treatment with tafamidis that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.

Why the committee made these recommendations

ATTR-CM can lead to heart failure, but treatment options are limited to managing symptoms and best supportive care. Awareness of ATTR-CM has improved, but accurately diagnosing ATTR-CM can be challenging and can take a long time.

Tafamidis is the first treatment for ATTR-CM that aims to treat the disease. Evidence from clinical trials shows that it reduces deaths and hospitalisation from conditions affecting the heart and blood vessels compared with placebo. But clinical benefit varies across different types and stages of ATTR-CM. Also, the measure used to assess how severe ATTR‑CM is has limitations. This makes it difficult to clearly identify who benefits from tafamidis and whether they should continue treatment.

The cost-effectiveness estimates are higher than what NICE normally considers an acceptable use of NHS resources. This is because there is not enough evidence that recommending tafamidis would reduce diagnosis delays and uncertainty about how long the treatment works after it is stopped. So, tafamidis is not recommended.