First Treatment for Rare Heart Condition Accepted for Use by Scottish Medicines Consortium
Pfizer Ltd have announced that Vyndaqel (tafamidis) is accepted for use within NHS Scotland for the treatment of wild-type and hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
Tafamidis is the first treatment for wild-type or hereditary ATTR-CM and the SMC is the first reimbursement body within the UK to make the treatment routinely available on the NHS.
ATTR-CM is an underdiagnosed and potentially fatal disease of the heart muscle. In ATTR-CM, a protein called transthyretin that normally circulates in the bloodstream becomes misshapen and the build-up of these proteins form amyloid in the heart2 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. As the condition progresses, the heart can become unable to adequately squeeze to pump blood out of the heart, ultimately leading to heart failure.
There are two types of ATTR-CM. Hereditary ATTR-CM (hATTR-CM), where there is a variant in the transthyretin gene which runs in families. Symptoms may start as early as age 20 and as late as age 80. The second type is wild-type ATTR-CM (wtATTR-CM), in which there is no variant in the transthyretin gene and symptoms usually start after age 65.
Dr Caroline Coats, Consultant Cardiologist, NHS Greater Glasgow and Clyde said: “Transthyretin amyloid cardiomyopathy is a rare, under-diagnosed and potentially life-threatening condition, for which there is a significant unmet medical need. This decision is a welcome step to help expand treatment options for patients.”
ATTR-CM is significantly under or misdiagnosed, making it difficult to characterise worldwide prevalence. The exact prevalence of ATTR-CM is unknown, but it is estimated that just under 100 adults in Scotland have this condition. Once diagnosed, the median life expectancy in people with ATTR-CM, dependent on sub-type, without treatment, is approximately 2 years to 5.5 years.
Joel Rose, CEO, Cardiomyopathy UK commented: “ATTR-CM significantly impacts everyday life for an individual and progressively worsens over time. Therefore, we’re pleased there is now an approved treatment option for eligible patients living in Scotland which could help improve their care.”
Tafamidis is currently being assessed by the National Institute for Health and Care Excellence (NICE) with a decision expected in 2024.