NHS Offers New Hope for 1,500 Heart Patients

Vutrisiran, a novel at-home injection, is now accessible through the NHS, marking a significant development for an estimated 1,500 patients in England and Wales diagnosed with transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This rare and progressive heart condition stems from misfolded liver-produced transthyretin proteins, which deposit and stiffen the heart muscle, often leading to severe symptoms like shortness of breath and chest pain.

The newly approved treatment, marketed as Amvuttra and developed by Alnylam Pharmaceuticals, operates by targeting and inhibiting messenger RNA (mRNA). This mechanism effectively reduces the liver's production of transthyretin. Administered every three months in the comfort of a patient's home, vutrisiran has been recommended by Nice as a valuable therapeutic option for eligible adults with ATTR-CM, offering a crucial alternative to existing treatments.

This advancement is hailed by patient advocacy groups and medical professionals as a vital step forward. It provides much-needed hope and improved quality of life for individuals and families affected by ATTR-CM. While tafamidis was the first treatment approved for NHS use in 2024, vutrisiran offers similar efficacy in prolonging life and slowing disease progression, at a comparable or lower cost, and with the added convenience of self-administration.