tafamidis (Vyndaqel)

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

tafamidis (Vyndaqel®) is accepted 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.

This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.

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