With the determination of the President no. 655/2026 (Rome, 21 May 2026; published in the Official Gazette on 28 May 2026), the Italian Medicines Agency extended the reimbursability of vutrisiran to the treatment of theamyloidosis from transthyretin wild-type or hereditary with cardiomyopathy (ATTR-CM) in adults. This is the first therapy with RNA interference (RNAi) mechanism reimbursed by the National Health Service for this indication, in a clinical setting so far managed by TTR tetramer stabilizers and heart failure management.
Transthyretin: from physiology to amyloid deposition
The transthyretin (TTR) is a tetrameric plasma protein synthesized predominantly by the liver (to a lesser extent by choroid plexuses and retinal pigmented epithelium). It performs the function of transporting thyroxine (from which the acronym trans-thy-retin) and, especially, of retinol carried by retinol-binding protein 4 (RBP4): TTR is in fact the main circulating carrier of vitamin A.