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.
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- 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
- Date advice published
- 13 November 2023