In a significant move for patient care, the NHS has approved a new at-home treatment for a rare and debilitating heart condition. The decision promises to transform the lives of approximately 1,500 people across England and Wales.
A Breakthrough for a Rare Heart Condition
The National Institute for Health and Care Excellence (Nice) has given the green light for the use of vutrisiran, a drug that treats transthyretin amyloidosis with cardiomyopathy (ATTR-CM). This progressive illness occurs when a protein produced by the liver, called transthyretin, misfolds. These misfolded proteins build up in the heart, causing it to stiffen and leading to symptoms like shortness of breath, chest pain, and swelling in the legs. Without treatment, the condition often results in heart failure.
The new treatment, branded as Amvuttra and manufactured by Alnylam Pharmaceuticals, offers a novel approach. It works by targeting and suppressing messenger RNA (mRNA) to reduce the liver's production of the problematic transthyretin protein. A key benefit for patients is the method of administration: the injection is taken at home once every three months, providing greater flexibility and independence.
Transforming Patient Lives and Treatment Options
Medical professionals and patient advocacy groups have welcomed the approval as a landmark moment. Anna Tomlinson, chief executive of Cardiomyopathy UK, described ATTR-CM as a "devastating, progressive condition" that frequently goes undiagnosed for years. She stated that this new option is "vitally important" and marks an "important moment" for improving the lives of those affected and their families.
This is not the first treatment for ATTR-CM available on the NHS. A daily tablet called tafamidis was approved in 2024. However, evidence reviewed by Nice suggests that vutrisiran helps patients live for a similar length of time and slows the progression of the disease at a comparable rate to tafamidis. Crucially, the cost of vutrisiran is similar to or lower than the existing treatment, making it a cost-effective alternative.
Expert Reactions and Future Hope
Phil Davey, UK and Ireland country manager for Alnylam Pharmaceuticals, expressed delight at the decision, noting that for decades, patients have faced limited options. He hopes vutrisiran will provide greater choice for cardiologists and improve patient outcomes.
Professor Bryan Williams from the British Heart Foundation echoed this optimism, calling the approval "very promising." He emphasised that effective treatments for this form of heart failure have been long-awaited and that this decision marks an important moment that could lead to fewer deaths and a better quality of life.
Adding to the chorus of support, Professor Simon Ray, national clinical director for heart disease at NHS England, highlighted the innovative nature of the therapy. He pointed out that it is based on Nobel-prize winning research and offers a welcome alternative to daily pills for some patients, all while being deliverable at home in the longer term.
