Disease-modifying treatment accepted for patients with ATTR-CM

The Scottish Medicines Consortium (SMC) has accepted the first long-acting disease-modifying treatment for the progressive heat condition, ATTR-CM, for use in eligible patients.

ATTR-CM is a rare, progressive and life-limiting condition that affects approximately 1,500 patients in the UK, although the true number is likely to be higher due to underdiagnosis. It occurs when transthyretin (TTR), a type of protein, accumulates in the heart, forming amyloid deposits that cause cardiomyopathy.

Vutrisiran was also approved by NICE in December 2025. It is a subcutaneous injection, administered every three months, either by patients themselves, a healthcare professional or a carer.

It tackles the root cause of the condition, neutralising TTR, and has been shown to reduce all-cause mortality and recurrent cardiovascular events in patients with ATTR-CM. Vutrisiran is also indicated for hereditary transthyretin amyloidosis with polyneuropathy.

General practice nurses have an important role to play in recognising potential ATTR-CM in undiagnosed patients. The disease often appears through a series of small clues that occur in different body systems, over several years, including reduced walking capacity or ability to manage usual activities, peripheral neuropathy, or carpal tunnel syndrome. Patients may have been referred to multiple specialists before someone ‘connects the dots’ and recognises the common underlying cause.

GPNs also have a role in the care of patients who have been prescribed vutrisiran, which can cause vitamin A deficiency: they should be prescribed a vitamin A supplement and referred to an ophthalmologist if they develop ocular symptoms such as night blindness.

Read our recent article on ATTR-CM to find out more.