New jab which could help thousands with rare heart condition approved for NHS use
A new at-home injection has been approved for use on the NHS offering a significant breakthrough for approximately 1,500 individuals in England and Wales living with a rare heart condition.
The treatment, administered every three months, received the green light from the National Institute for Health and Care Excellence (Nice).
Vutrisiran will be available for patients suffering from transthyretin amyloidosis with cardiomyopathy (ATTR-CM), a debilitating illness where the liver-produced protein transthyretin misfolds, leading to deposits that stiffen the heart.
Patients typically experience symptoms such as shortness of breath, chest pain, and swelling in the lower legs.
Without intervention, this progressive condition can tragically culminate in heart failure.
Vutrisiran, sold under the brand name Amvuttra and made by Alnylam Pharmaceuticals, works by binding to, and stifling messenger RNA (mRNA) to reduce the amount of transthyretin made by the liver.
The injection, taken every three months by patients in their own home, has been recommended by Nice as a treatment option for some adults with ATTR-CM.
Anna Tomlinson, chief executive of Cardiomyopathy UK, said: “ATTR-CM is a devastating, progressive condition that often goes undiagnosed for months, or even years.
“It may not only lead to heart failure, but also seriously impact a person’s daily life.
“New treatment options are therefore vitally important, and we welcome Nice’s decision to make vutrisiran available on the NHS across England and Wales.
“This marks an important moment that we hope will further improve the lives of those affected and their families.”
Phil Davey, country manager at Alnylam Pharmaceuticals, UK and Ireland, said: “For decades, people living with this devastating cardiac disease have faced limited treatment options.
“The team at Alnylam are delighted by the final draft guidance issued by Nice and the recognition of the important role vutrisiran can play in the NHS care of those impacted.
“Our hope is that vutrisiran will support greater choice and flexibility for cardiologists and ultimately improve outcomes for patients.”
The first treatment for ATTR-CM – a once-a-day tablet called tafamidis – was approved for NHS use in 2024.
According to Nice, evidence suggests vutrisiran helps people live for about as long as those on tafamidis, and that vutrisiran delays the condition getting worse at a similar rate.
The cost of vutrisiran is also similar to or lower than tafamidis.
Professor Bryan Williams, chief scientific and medical officer at the British Heart Foundation, said: “Transthyretin amyloidosis causes progressive heart failure, which can be devastating for patients and their families.
“We have been searching for effective treatments for a long time and at last they have begun to emerge.
“Nice’s decision to make vutrisiran available on the NHS across England and Wales is very promising and marks an important moment in the treatment landscape, which we hope will lead to a significant reduction in the number of deaths and further improve quality of life for those with the condition.”
Professor Simon Ray, national clinical director for heart disease at NHS England, added: “This innovative therapy – which is based on Nobel-prize winning research and can be delivered at home in the longer term – will also offer an alternative to current daily pills for some, and we are delighted that NHS patients will now be able to benefit from it.”
