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.

Andrew Stephenson, Health Minister 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”.

Tafamidis works by slowing the build-up of dangerous protein deposits, with clinical trials showing a 41% 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.

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”.

Peter Salussolia, an 80 year-old from South West London and chairman of a hotel and leisure group was diagnosed with ATTR-CM in 2018. He received tafamidis as part of an early access scheme at The Royal Free Hospital.

Peter said: “Since taking tafamidis, I have seen no deterioration in my condition and if anything, my condition is stable and slightly improved. It truly has been amazing after 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”.

The NHS has rolled out a number of breakthrough drugs this year, including a new immunotherapy for women with advanced endometrial cancer, and a targeted combination treatment that can significantly slow the progress of brain tumours in children.

Cardiomyopathy UK CEO Joel Rose said: “ATTR-CM has significant impacts on an individual’s everyday life and we are really pleased there is now a treatment option which could help improve their care. We now must make sure that this treatment can get to those who will benefit as quickly as possible as ATTR-CM often worsens over time”.

Susan Rienow, Country President, Pfizer UK said: “This positive NICE decision, and interim funding through the Innovative Medicines Fund, is a significant milestone for eligible patients. We’ve remained committed to ensuring access to tafamidis and we’re pleased that it will now be available across the UK. We hope this step will contribute towards further improving the diagnosis and care of people with transthyretin amyloid cardiomyopathy”.

Source: NHS / Public Health England


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