We welcome the decision by the Scottish Medicines Consortium (SMC) to accept vutrisiran for use within NHS Scotland for adults living with transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
What is ATTR-CM?
ATTR-CM is a rare, progressive and life-limiting condition. Abnormal proteins called amyloid build up in the heart muscle, making it stiff and less able to pump blood around the body. People living with ATTR-CM often have fatigue, breathlessness, swelling and problems with mobility – and the damage to the heart it causes can’t be reversed.
Vutrisiran (brand name Amvuttra) is a disease-modifying therapy that targets the underlying cause of ATTR-CM. It reduces the production of the transthyretin (TTR) protein that forms amyloid deposits. It’s given as an injection about once every three months.
Scotland follows on from England and Wales
The SMC’s decision last month means that people in Scotland can now be offered the same treatment that NICE recommended for use in England and Wales last year.
The SMC has accepted vutrisiran as a monotherapy. This means it can be used on its own, but not together with other disease-modifying treatments for ATTR-CM, such as tafamidis. If you have ATTR-CM, your specialist team can tell you which treatment option is right for you.
Dr Rob Peel, Chair of the SMC, said that vutrisiran “offers another treatment option for patients with ATTR-CM, that is administered in a different way from the currently available option and may suit some patients better.”
Bringing patient voices to the decision
The SMC advises NHS Scotland on how clinically effective and cost-effective new medicines are. Most new medicines have to be approved by the SMC before they can be routinely used in the NHS in Scotland.
Understanding how a new medicine affects the quality of life of patients and carers is an important part of how the SMC makes its decisions. As part of the assessment, Cardiomyopathy UK submitted insights from our patient community and clinical nurse specialists.
These insights showed how living with ATTR-CM, or caring for someone who does, affects people’s ability to exercise, their mobility, their self-confidence and their mental health. They also covered people’s experiences of current treatment and care, and what a new treatment option could mean for eligible patients.
Find out more
If you have ATTR-CM, you’re being tested for possible ATTR-CM, or you have questions about what this decision means for you, our specialist nurses can give general advice and signpost you to further support. Call 0800 018 1024 to speak to a member of the team (see helpline opening hours here).
For more specific advice, please speak with your specialist clinical team.