New Option for ATTR-CM
ATTR-CM is a rare, fatal condition in which transthyretin (TTR) amyloid deposits stiffen the heart and lead to heart failure. TTR is produced in the liver, and amyloid can also accumulate in the peripheral nerves and gastrointestinal tract.
Wild-type ATTR-CM arises when TTR becomes structurally unstable with age. Hereditary ATTR-CM is caused by one of more than 120 TTR gene mutations.
Tafamidis, an oral TTR stabiliser, is the only therapy currently accepted for use in Scotland for wild-type and hereditary ATTR-CM. Other management approaches include diuretics for congestive symptoms and treatments for arrhythmias and conduction abnormalities. Cardiac or liver transplantation may be considered in selected patients, but advanced age and comorbidities mean transplantation is rarely an option.
Vutrisiran is a small interfering RNA therapy that targets and suppresses hepatic synthesis of both wild-type and variant TTR protein via RNA interference. By lowering circulating TTR levels, it reduces tissue TTR amyloid deposition.
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