First ever life-saving treatment for rare heart condition available on NHS



NHS patients with a life-threatening heart condition are set to benefit from a cutting-edge new medicine which can significantly reduce the risk of hospitalisation and death.

The drug, tafamidis, is the first ever approved treatment for a cohort of patients in England with a rare heart condition known as transthyretin amyloidosis cardiomyopathy (ATTR-CM), where clumps of protein build up in the heart, often resulting in heart failure and potentially proving fatal.

People living with the progressive condition will now be able to benefit from the once-a-day capsule following a recommendation to offer the innovative treatment to more than one thousand eligible patients.

Professor 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 – with NHS patients now able to benefit from a once-a-day treatment that can reduce the risk of hospitalisation and heart failure.

“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”.

Tafamidis works by slowing the build-up of dangerous protein deposits, with clinical trials showing a 41 per cent reduction in the risk of death in patients taking the treatment compared to patients on a placebo.

Patients receiving tafamidis through clinical trials also experienced fewer hospitalisations due to their condition than the placebo group.

The drug is available on the NHS from today (13 May) thanks to interim funding from the Innovative Medicines Fund after the National Institute of Health and Care Excellence (NICE) gave the treatment the green light.

Around 1,500 people across England have been diagnosed with ATTR-CM.

Everyday symptoms of the condition can include shortness of breath, palpitations and abnormal heart rhythms, fatigue, fainting and chest pain.

Previous treatment options for ATTR-CM have been limited and mainly focus on symptom management and supportive care.

Heath Minister, Andrew Stephenson, said: “We’re working to make healthcare faster, simpler and fairer for everyone, including people with very rare conditions such as this.

“Thanks to the Innovative Medicines Fund, patients with this life-threating condition will be able to access the new medicine on the NHS immediately at a price that is fair to the taxpayer”.

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