NHS rolls out first-ever treatment for patients with rare type of cardiomyopathy

Patients with a rare heart condition known as transthyretin amyloidosis cardiomyopathy are set to benefit from the first approved treatment for the disease.

More than 1,000 patients will be eligible for the treatment which is now available on the NHS following an approval from the National Institute of Health and Care Excellence (NICE).

Clinical trials show tafamidis reduces mortality risk and hospitalisations

Tafamidis (Vyndamax) is a transthyretin stabiliser which inhibits amyloid formation (the build-up of protein deposits), thereby delaying the development of nerve and cardiac muscle damage caused by transthyretin amyloidosis.

The drug comes in the form of a capsule and is taken orally once a day. It has been shown to reduce disease progression and mortality with clinical trials showing that tafamidis reduced the risk of death by 41% compared to placebo. Patients taking the drug also experienced fewer hospitalisations than the control group.

Peter Salussolia, an 80 year-old from South West London, was diagnosed with transthyretin amyloidosis cardiomyopathy 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”.

Cardiomyopathy UK has welcomed the first and only treatment option for ATTR-CM

Around 1,500 people in England have a diagnosis of transthyretin amyloidosis cardiomyopathy, a rare but severe cause of restrictive cardiomyopathy, caused by the accumulation of transthyretin fibrils in the myocardium.

Transthyretin amyloidosis cardiomyopathy can be hereditary, caused by an inherited mutation in the TTR gene, or it can occur spontaneously.

Symptoms include fatigue, dyspnoea, palpitations and abnormal heart rhythms, fainting and chest pain. Symptom onset typically occurs in midlife, although some patients may not have symptoms until much later.

Cardiomyopathy UK has welcomed the roll out of the new treatment, which is set to benefit more than 1,000 patients.

Joel Rose, CEO of the charity, 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”.