following a full submission 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.
The licence holder has indicated their intention to resubmit.
- Medicine name:
- tafamidis (Vyndaqel)
- SMC ID:
For the treatment of wild‑type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM)
- Pharmaceutical company
- Pfizer Ltd
- BNF chapter
- Cardiovascular system
- Submission type
- Not recommended
- Date advice published
- 12 July 2021