The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending Amvuttra (vutrisiran) for adults with transthyretin amyloidosis with cardiomyopathy (ATTR-CM) in England and Wales.
Vutrisiran is a subcutaneous injection that can be self-administered every three months. Around 1,500 patients across England and Wales could be eligible for the treatment, which is the first and only long-acting disease-modifying therapy to treat both ATTR-CM and hereditary ATTR with stage 1 or 2 polyneuropathy.
Alnylam UK Limited welcomed the decision, noting that final guidance is expected in December 2025. Vutrisiran works by reducing the production of transthyretin proteins, including unstable, misfolded proteins that cause damage to the heart. Clinical data from the HELIOS-B study showed reductions in all-cause mortality and recurrent cardiovascular events compared with placebo, as well as fewer hospital visits.
Marianna Fontana, Professor of Cardiology at University College London and HELIOS-B investigator, said: “Today’s decision from NICE will be hugely welcomed news for patients and cardiologists. ATTR-CM – or ‘stiff heart disease’, as it is sometimes referred to – is the result of the body producing proteins which can change in structure and ultimately cause life-threatening damage to the heart.”
She added: “For the first time in ATTR-CM, we have a medicine that works right at the start of this process, preventing these proteins from ever being produced. Having an effective, long-acting medicine that can be taken at home every three months, is a real step forward in the management of this disease.”
Anna Tomlinson, Chief Executive Officer at Cardiomyopathy UK, explained: “ATTR-CM is a devastating, progressive condition that often goes undiagnosed for months – or even years. New treatment options are therefore vitally important, and we welcome NICE’s decision to make vutrisiran available on the NHS across England and Wales.”
