We welcome the positive Final Draft Guidance from the National Institute for Health and Care Excellence (NICE), recommending vutrisiran, a new long-acting treatment for adults living with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in England and Wales.
ATTR-CM is a rare, progressive and life-limiting condition that often goes unrecognised for long periods. Many people experience years of symptoms - including fatigue, breathlessness, swelling and mobility problems - before receiving a diagnosis. During this time, irreversible damage to the heart can occur, significantly affecting quality of life for individuals and their families.
Vutrisiran is the first long-acting, disease-modifying therapy that targets the underlying cause of ATTR-CM. It is administered as a subcutaneous injection, usually every three months, and can commonly be self-administered at home. This means that eligible patients may have access to a treatment that fits more easily around everyday life, potentially reducing hospital visits and easing pressure on NHS services. Approximately 1,500 people across England and Wales could be eligible.
A meaningful step forward for patients
We know through our helpline, support groups and patient community that many individuals with suspected or confirmed ATTR-CM struggle to access timely diagnosis, specialist care and consistent treatment options. Today’s recommendation is therefore an important milestone, offering hope of improved stability, slowed disease progression, and better day-to-day wellbeing.
Professor Marianna Fontana, Professor of Cardiology at University College London and the National Amyloidosis Centre, described the decision as “hugely welcomed news for patients and cardiologists,” noting the significance of a medicine that works at the beginning of the disease process and can be taken at home every three months.
Anna Tomlinson, Chief Executive of Cardiomyopathy UK said:
“For many people, ATTR-CM affects every part of daily life, yet it is still too often overlooked or diagnosed far too late. We regularly hear from individuals who have spent years searching for answers as their symptoms steadily worsen.
NICE’s decision to recommend vutrisiran is therefore a hugely welcome development. It offers a much-needed treatment option that may help slow the progression of this devastating condition and give people greater stability, confidence and control.
This represents an important milestone for those living with ATTR-CM and their families. We now hope to see continued progress in expanding the amyloidosis networked model of care, ensuring that as many patients as possible are able to access timely diagnosis and treatments such as this.
As the national charity supporting people with cardiomyopathy, we see this as a step towards wider improvements—better diagnostic pathways, stronger access to specialist services, and the support families need to navigate such a complex condition.”
Ensuring people can access the treatment they need
While this recommendation is excellent news, further work is still required to ensure equitable access across all regions. Cardiomyopathy UK will continue to work with patients, clinicians, NHS partners and policymakers to:
- improve awareness of the signs and symptoms of ATTR-CM and other cardiomyopathies
- support earlier and more accurate diagnosis
- address regional variations in access to specialist services
- ensure patients and families receive the information and support they need