1 Recommendations

1.1

Vutrisiran can be used, within its marketing authorisation, as an option to treat wild-type or hereditary transthyretin amyloidosis with cardiomyopathy in adults. Vutrisiran can only be used if the company provides it according to the commercial arrangement.

1.2

Use the least expensive option of the suitable treatments (including vutrisiran and tafamidis), having discussed the advantages and disadvantages of the available treatments with the person with the condition. Take account of administration costs, dosages, price per dose and commercial arrangements.

What this means in practice

Vutrisiran must be funded in the NHS in England for the condition and population in the recommendations, if it is considered the most suitable treatment option. Vutrisiran must be funded in England within 90 days of final publication of this guidance.

There is enough evidence to show that vutrisiran provides benefits and value for money, so it can be used routinely across the NHS in this population.

Why the committee made these recommendations

Usual treatment for transthyretin amyloidosis with cardiomyopathy is tafamidis.

The clinical trial was not designed to directly compare vutrisiran with tafamidis, and the results from indirect comparisons are uncertain. But the results from both analyses suggest that people having vutrisiran live for about as long as people having tafamidis, and that vutrisiran delays the condition getting worse at a similar rate as tafamidis.

The costs for vutrisiran are similar to or lower than for tafamidis. So, because the clinical evidence suggests that vutrisiran is likely to work as well as tafamidis, vutrisiran can be used.