Tafamidis approved by NICE

News 12 May 2024

The National Institute for Health and Care Excellence (NICE) thee organisation that advises which medicines should be made available via the NHS in England has given their approval to a new drug – tafamidis (Vyndaqel) – to treat transthyretin amyloidosis cardiomyopathy (ATTR-CM). This is the first ever treatment for this type of cardiomyopathy. It has been approved for adults with hereditary or wild-type (non-hereditary) ATTR-CM. The approval follows that of Scotland where the treatment has been available since November 2023.

In response to the news, Joel Rose, Cardiomyopathy UK’s Chief Executive, said:

“Amyloidosis cardiomyopathy is a serious, progressive, disabling and lethal condition, with a heavy toll on patients, their relatives and carers. So we’re extremely pleased that Tafamidis will become available more widely in the UK for people affected by this rare form of cardiomyopathy".

How does the treatment work?

Amyloidosis is a group of rare conditions caused by a build-up of an abnormal protein (called amyloid) in the body. This build-up stops the tissues and organs working properly.

In ATTR-CM, a type of amyloid protein called transthyretin that normally circulates in the bloodstream becomes misshapen and builds up in the heart, nerves and other organs. It can affect how well the heart functions, causing cardiomyopathy.

Tafamidis works by inhibiting the formation of amyloid. This delays the onset of cardiac muscle damage.

Our role

We worked closely with the Amyloidosis UK to submit evidence to NICE. This submission provided evidence on the impact of the disease on patients, and how the drug could make a difference in improving the quality of life of patients, their family and carers.

We also spoke Pfizer – the pharmaceutical company which owns the drug - about the importance of making the drug affordable to the NHS.

Next steps

It is likely that it will take some time for the NHS in England, Wales and Northern Ireland to put in place the processes needed to prescribe and monitor this new treatment. We will work with all the key stakeholders to help ensure that anyone who is likely to benefit from Tafamidis will be access it as soon as possible.

More broadly, as new treatments are being developed the charity will continue to work with NICE, the Scottish Medicines Consortium and pharmaceutical companies to help them understand the experience of living with cardiomyopathy and to promote the development and use of effective and affordable treatment options.

You can read the full report from the NICE here.

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