tafamidis (Vyndaqel)

following a resubmission assessed under the orphan and end of life medicine process:

tafamidis (Vyndaqel®) is not recommended for use within NHSScotland.

Indication under review: for the treatment of wild-type and hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
In a phase III study, 30 months of treatment with tafamidis (as meglumine) significantly reduced the risk of all-cause mortality and cardiovascular-related hospitalisation compared with placebo, in patients with wild-type or hereditary ATTR-CM.

The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust economic analysis to gain acceptance by SMC.

This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.​