Pfizer’s Vyndaqel (tafamidis) has been recommended by the National Institute of Health and Care Excellence (NICE) to treat transthyretin amyloidosis cardiomyopathy (ATTR-CM), a rare heart disease affecting about 1,500 people in England.
The drug has been specifically recommended for use in adults with wild-type or hereditary forms of the condition and is now the first ATTR-CM therapy to become available via the NHS.
In ATTR-CM, the transthyretin protein that normally circulates in the bloodstream becomes misshapen and accumulates in various parts of the body, including the heart. This causes the heart muscle to stiffen over time, eventually leading to heart failure.
The median life expectancy in untreated patients with ATTR-CM is approximately two to 3.5 years after diagnosis, with previous treatment options for the disease mainly focusing on symptom management and supportive care.
Vyndaqel, taken as a once-daily capsule, works by slowing the build-up of TTR and was associated with a significant reduction in the risk of death and ATTR-CM-related hospitalisations compared to placebo in clinical trials.
The drug has been made available on the NHS immediately following interim funding from the Innovative Medicines Fund, which facilitates faster access to new treatment while further data is collected, and is expected to benefit more than 1,000 eligible patients.
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 hope this step will contribute towards further improving the diagnosis and care of people with ATTR-CM.”
Also commenting on NICE’s decision, professor Simon Ray, national clinical director for heart disease at NHS England, said: “A first of its kind, [Vyndaqel] 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.”
The authorisation comes just over a week after NICE recommended Pfizer’s Oxbryta (voxelotor) to treat sickle cell disease in patients aged 12 years and older, with the therapy also being funded immediately through the Innovative Medicines Fund.
