Although we will not be issuing a
formal press release, we wanted to let you know that today (21
November 2025) NICE has issued final draft guidance which
recommends vutrisiran (also called Amvuttra and made by
Alnylam Pharmaceuticals) as an option for some adults with
transthyretin amyloidosis with cardiomyopathy
(ATTR-CM).
In England, around 1,200 people have
been diagnosed with ATTR-CM, a progressive,
life-limiting and debilitating condition that causes
the liver to produce abnormal transthyretin protein. These
abnormal proteins deposit as amyloids in the
tissues of the body (amyloidosis). These can disrupt the
structure and damage the function of the affected
tissues.
When these deposits affect
the heart they can cause symptoms of cardiomyopathy such as
chest pain, shortness of breath, palpitations and abnormal
heart rhythms,
most frequently atrial fibrillation or atrial
flutter.
NICE already recommends tafamidis
as an option for people with ATTR-CM. The clinical
evidence suggests 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 recommended as
an additional treatment option.
Vutrisiran is an RNA interference
(RNAi) therapeutic that inhibits the production of
disease-causing transthyretin (TTR) protein by the liver, leading
to a reduction in the level of TTR in the blood. It
is given by injection under the skin (it can be
self-administered at home) every 3
months.
Vutrisiran costs £95,862.36 per
injection. At the recommended dose the estimated annual
cost per patient is £383,449.44. The company has a confidential
commercial arrangement that makes vutrisiran available
to the NHS with a discount.
Ends
Notes to
editors
-
The final draft guidance on
vutrisiran for treating
transthyretin amyloidosis
with cardiomyopathy is available on the NICE website (from
00:01, Friday, 21 November 2025) at: https://www.nice.org.uk/guidance/indevelopment/gid-ta11598/documents
