Although we will not be issuing a formal press release we wanted
to let you know that today (13 May 2024) NICE has published final
draft guidance which recommends tafamidis (also called Vyndaqel
and made by Pfizer) for treating transthyretin amyloidosis with
cardiomyopathy (ATTR-CM).
A new price deal agreed with the company, together with new
analyses looking at what happens after people stop taking
tafamidis, means that tafamidis can now be recommended for NHS
use as an option for adults with wild-type or hereditary ATTR-CM.
Today's announcement marks the latest stage of NICE's review of
its previous guidance published in 2021 that did not recommend
the treatment. Earlier draft guidance issued for public
consultation as part of this review also concluded that the
treatment could not be recommended as a cost-effective use of NHS
resources.
Around 1,500 people in England with ATTR-CM could now be eligible
for the treatment.
A progressive, life limiting and debilitating condition, ATTR-CM
is characterised by the abnormal build-up of a
protein called amyloid, primarily in the
heart. Symptoms start in adulthood and get worse over
time,causing the heart tissue to thicken and stiffen.
ATTR-CM can lead to heart failure, but current treatment options
are limited to managing symptoms and best supportive care, such
as diuretics.
Taken as a once-a-day tablet, tafamidis is the first treatment
for ATTR-CM that aims to treat the disease.
