- Author, Michelle Roberts
- Role, Digital health editor, BBC News
Patients with a rare but potentially deadly “stiff heart” condition can get a new once-a-day pill treatment on the NHS in England from Monday.
Tafamidis slows the build-up of risky proteins in the heart muscle of people with amyloidosis cardiomyopathy (ATTR-CM).
Experts say it should save lives, and trials suggest it can cut death risk by about 41%.
The drug is already approved in Scotland and draft guidelines for England and Wales now recommend it too.
Life-saving
Tafamidis, also called vyndaqel and made by Pfizer, is the first drug for ATTR-CM that aims to treat the cause of the condition.
Until now, options have been limited to managing symptoms and providing supportive care, although some NHS patients, including 80-year-old Peter Salussolia, have already been receiving tafamidis.
He said: “I was initially told my condition had no treatment and I was given around five years plus to live.
“This rollout offers a great beacon of hope for NHS patients who live with this debilitating and progressive condition. Personally, I have been able to continue skiing and boating and I am truly grateful for the effect that tafamidis has had on my condition.”
About 1,500 people in England with ATTR-CM could now be eligible for the treatment.
This rare heart condition typically starts to show in adulthood and gets worse over time as the heart tissue thickens and stiffens.
- shortness of breath
- palpitations and abnormal heart rhythms
- ankle swelling, fatigue, fainting and chest pain
Ultimately, it can cause heart failure.
Prof Simon Ray, national clinical director for heart disease at NHS England, said: ”A first of its kind, tafamidis will give those living with this rare progressive condition new hope.
“This pioneering drug is just one example of the NHS delivering on its commitment to ensure patients across the country have access to the latest and most effective treatments to help significantly improve their quality of life.”
The list price for a packet of 30 capsules of the drug is about £10,000, but Pfizer has a commercial agreement for NHS use which will be less, although the cost has not been made public.
Sarah Carter is a health and wellness expert residing in the UK. With a background in healthcare, she offers evidence-based advice on fitness, nutrition, and mental well-being, promoting healthier living for readers.