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NICE recommends first treatment for rare heart condition

The National Institute for Health and Care Excellence (NICE) has published final draft guidance which recommends Vyndaqel®▼ (tafamidis) for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM)1 to be routinely available on the NHS.

The NICE final draft guidance is subject to appeal, with final guidance anticipated in June. However, from today, patients in England will be able to access tafamidis via interim funding through the Innovative Medicines Fund (IMF), until it becomes routinely available on the NHS.

ATTR-CM is an underdiagnosed and potentially fatal disease of the heart muscle.2
In ATTR-CM, a protein called transthyretin that normally circulates in the bloodstream becomes misshapen and the build-up of these proteins cause amyloid deposits in the heart.2
The amyloid causes the walls of the heart to become stiff and the left ventricle is then unable to properly relax or fill with blood – this leads to cardiomyopathy.2 As the condition progresses, the heart can become unable to adequately squeeze to pump blood out, ultimately leading to heart failure.2

There are two types of ATTR-CM. The first is hereditary ATTR-CM (hATTR-CM), where there is a variant in the transthyretin gene which runs in families.2 Symptoms may start as early as 20 years old and as late as 80 years old.2 The second is wild-type ATTR-CM (wtATTR-CM), in which there is no variant in the transthyretin gene and symptoms usually start after age 65.2

The exact prevalence of ATTR-CM is unknown, but it is estimated that in England and Wales around 1000 people have been diagnosed with ATTR-CM.3,4
Once diagnosed, the median life expectancy in people with ATTR-CM, dependent on sub-type and without treatment, is approximately 2 years to 5.5 years.5,6

Tafamidis is the first treatment for wild-type or hereditary ATTR-CM to be routinely available on the NHS.

The Scottish Medicines Consortium (SMC) accepted tafamidis for use with adult patients with ATTR-CM within NHS Scotland in November 2023.7

Joel Rose, CEO, Cardiomyopathy UK said: “ATTR-CM has a significant impact on an individual’s everyday life, and we are really pleased there is now a treatment option available. We now must make sure that this treatment can get to those who will benefit as quickly as possible, as ATTR-CM often worsens over time.”

Susan Rienow, Country President, Pfizer UK said: “This positive NICE decision, and interim funding through the Innovative Medicines Fund, is a significant milestone for eligible patients. We’ve remained committed to ensuring access to tafamidis and we’re pleased that it will now be available across the UK. We hope this step will contribute towards further improving the diagnosis and care of people with transthyretin amyloid cardiomyopathy.”


1 National Institute for Health and Care Excellence (NICE). Tafamidis for treating transthyretin amyloidosis with cardiomyopathy. Available at: Overview | Tafamidis for treating transthyretin amyloidosis with cardiomyopathy | Guidance | NICE Last accessed: May 2024.

2 American Heart Association. Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Available at: https://www.heart.org/en/health-topics/cardiomyopathy/what-is-cardiomyopathy-in-adults/transthyretin-amyloid-cardiomyopathy-attr-cm. Last accessed: May 2024

3 Pfizer Ltd data on file. PP-VYN-GBR-1187. Last accessed: May 2024

4 Pfizer Ltd data on file. PP-VYN-GBR-1335. Last accessed: May 2024

5 Maurer MS. et al. Addressing Common Questions Encountered in the Diagnosis and Management of Cardiac Amyloidosis. Circulation. 2017; 135: 1357-1377. Last accessed: May 2024

6 Witteles RM, Bokhari S, Damy T, et al. Screening for transthyretin amyloid cardiomyopathy in everyday practice. JACC Heart Fail 2019;7:709–16. Last accessed: May 2024

7 Scottish Medicines Consortium (SMC) Vyndaqel (tafamidis) SMC2585 November 2023. www.scottishmedicines.org.uk Last accessed: May 2024

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Joanna Harvey
Joanna Harvey
Marketing and Communication Executive | Uniphar Commercial


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