Alnylam UK Limited has welcomed a Scottish Medicines Consortium decision to accept vutrisiran for adults with ATTR‑CM, enabling eligible patients to access AMVUTTRA through NHS Scotland. The therapy is the first long‑acting disease‑modifying option to target the underlying biology of the condition.
Vutrisiran is administered as a subcutaneous injection every three months and delivers rapid, sustained knockdown of transthyretin proteins, observed from six weeks and persisting up to 30 months.
An estimated 1,800 people in Scotland could be living with ATTR‑CM, including diagnosed and undiagnosed patients. Vutrisiran is also indicated for hereditary ATTR amyloidosis in adults with stage 1 or 2 polyneuropathy.
Dr Joanne Simpson, Cardiologist, Queen Elizabeth University Hospital, Glasgow, said: “The SMC’s acceptance represents a significant step forward for patients and clinicians in Scotland. ATTR-CM is a progressive condition causing heart failure and reduced quality of life. Vutrisiran targets the underlying biology of the disease and represents an important additional disease modifying treatment option for eligible patients. This decision is meaningful progress in management of ATTR-CM, whilst continued efforts to improve early recognition, diagnosis and timely referral are essential.”
ATTR‑CM affects around 1,500 people across the UK, though under‑diagnosis means the true number is likely higher. It occurs when misfolded transthyretin proteins accumulate in the heart, forming amyloid deposits that stiffen the muscle and impair its ability to pump blood. If untreated, the disease is associated with high mortality.
Katharine McIntosh, Chief Executive Office at Cardiomyopathy UK, explained: “The SMC’s decision on vutrisiran is very welcome news for people living with ATTR-CM in Scotland. ATTR-CM is a rare, progressive and life-limiting condition that can affect physical health and mental wellbeing, and we were pleased to be able to share our patient community’s experiences as part of the assessment. Having another treatment option – one that targets the underlying cause, and that can often be self-administered at home – gives eligible patients greater choice.”
Kate Taylor, Chief Executive Office at Amyloidosis UK, said: “ATTR with cardiomyopathy can have a significant impact on people’s lives and on those closest to them. People can face a long journey to diagnosis, highlighting the importance of greater awareness and earlier recognition of the condition so that people can receive appropriate care as soon as possible.”
With this recommendation, vutrisiran becomes the first treatment accepted by the SMC for both ATTR‑CM and hereditary ATTR with stage 1 or 2 polyneuropathy. The decision follows NICE’s recommendation in England and Wales in December 2025.
Phil Davey, General Manager, Alnylam Pharmaceuticals, UK, Ireland and Canada, added: “We welcome the SMC’s decision, which represents an important milestone for people living with ATTR-CM in Scotland. At Alnylam, we are committed to translating the potential of RNAi into meaningful treatment options for patients, and this recommendation is an important step on that journey. We would like to thank the SMC, the patient community, clinicians and NHS Scotland for their continued collaboration in helping make this treatment available.”
