Scotland's NHS now has a second treatment option for transthyretin amyloid cardiomyopathy (ATTR-CM), a rare and fatal condition where protein deposits stiffen the heart and lead to heart failure. The Scottish Medicines Consortium (SMC) has approved vutrisiran (Amvuttra), an RNA interference (RNAi) therapy, for restricted use as monotherapy in adults with both wild-type and hereditary ATTR-CM — but only under a patient access scheme that guarantees cost-effectiveness.

Vutrisiran works by silencing the liver's production of the misfolded TTR protein, reducing amyloid buildup in the heart and other tissues. It's administered as a subcutaneous injection once every three months — a different delivery method from tafamidis, the only previously accepted therapy in Scotland. The approval is backed by the HELIOS-B phase 3 trial, which showed vutrisiran significantly reduced the risk of death and cardiovascular events compared to placebo.

By the Numbers:

• HR 0.67 for composite mortality/CV events in the monotherapy subgroup vs. placebo
• All-cause mortality at 42 months: 22% (vutrisiran) vs. 29% (placebo) in the monotherapy subgroup
• 6-minute walk distance decline slowed by ~26.5 m vs. placebo at 30 months
• Hereditary ATTR-CM patients made up only 12% of trial participants — a noted uncertainty

Why it matters: ATTR-CM is progressive and often diagnosed late, leaving patients with few options. Adding vutrisiran gives clinicians a novel mechanism of action and a quarterly dosing schedule that may better suit certain patients, expanding the toolkit for a condition where early treatment is critical to preserving heart function.