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A pioneering drug that targets a life-threatening heart condition is “a beacon of hope” for thousands of sufferers, says a businessman given five years to live.
Peter Salussolia, 80, from South West London, was diagnosed with transthyretin amyloidosis cardiomyopathy (ATTR-CM) in 2018.
The potentially fatal condition causes clumps of protein to build up in the heart, often resulting in heart failure.
But Peter’s condition is now stable after he began taking tafamidis, a daily pill that was found to cut patients’ risk of death by 41 percent in a clinical trial.
The treatment has been approved for widespread NHS use from Monday, benefitting up to 1,500 sufferers in England.
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Hotel and leisure group chairman Paul, who accessed the drug early through a scheme at The Royal Free Hospital, 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.”
Common symptoms of ATTR-CM include shortness of breath, palpitations and abnormal heart rhythms, fatigue, fainting and chest pain.
Until now, treatment options were limited and mainly focused on symptom management and supportive care.
Patients who took tafamidis in trials were also less likely to end up in hospital. Made by Pfizer, it works by slowing the build-up of dangerous protein deposits.
Professor Simon Ray, NHS England’s national clinical director for heart disease, 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.”
Joel Rose, CEO of charity Cardiomyopathy UK, 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.”
Health minister Andrew Stephenson said the approval was thanks to the Innovative Medicines Fund, which helps patients with life-threatening conditions access new medicines sooner.
He added: “We’re working to make healthcare faster, simpler and fairer for everyone, including people with very rare conditions such as this.”
