NICE rejects Pfizer’s Vyndaqel for treating rare heart condition
pharmafile | May 12, 2021 | News story | Research and Development | NICE, Pfizer, Vyandaqel
NICE has published final guidance today which rejects tafamidis, also known as Vyndaqel, for treating rare heart condition transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
Tafamidis is the first treatment for ATTR-CM that aims to treat the disease. However, from the clinical trials conducted it is difficult to establish which patients may benefit from the drug.
NICE is still keen to enable access to people ATTR-CM and will conduct a rapid review if NHS and Pfizer are able to show this is treatment is a cost-effective use of NHS resources.
The cost-effectiveness estimates are higher than what are usually accepted due to the lack of evidence that tafamidis would reduce delays in diagnosis and uncertainty about how long the benefit from the drug lasts after it is stopped.
For these reasons, tafamidis could not be recommended.
ATTR-CM can lead to heart failure, and current treatments, such as diuretics, are limited to managing symptoms and best supportive care.
This news follows an unsuccessful appeal against the draft recommendations by the company, Cardiomyopathy UK, and the British Society for Heart Failure.
There are around 800 people in the UK with ATTR-CM. It can be a life-limiting condition and is characterised by the abnormal build-up of a protein called amyloid in the heart. Symptoms start in adulthood and progressively get worse over time, causing the heart tissue to thicken and stiffen.
Reduced delays in diagnosis would allow earlier treatment in less advanced disease states as well as incurring less health-care costs and a reduction in patient anxiety.
Lilly Subbotin
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