Vutrisiran for treating transthyretin amyloidosis with cardiomyopathy

Vutrisiran is a medicinal product that is in development for the treatment of adults with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). ATTR-CM is classified as either wildtype transthyretin (wtATTR) amyloidosis with cardiomyopathy (wtATTR-CM) or hereditary transthyretin (hATTR) amyloidosis with cardiomyopathy (hATTR-CM). ATTR-CM occurs when a protein called transthyretin (TTR) becomes defective and aggregates, forming amyloid deposits in the heart and other organs. Amyloid deposits can damage the structure and function of the organs, leading to the thickening and stiffening of heart tissues (cardiomyopathy), which can cause severe disease and be fatal. The wtATTR form results from the normal functioning TTR protein becoming defective with ageing. In contrast, the hATTR form occurs due to a faulty gene that runs in families and can be passed down from biological parents to their children. ATTR-CM is a poorly recognised condition with delayed diagnosis and poor prognosis, and treatment options are limited to best supportive care to the management of symptoms.