NHS adopts Pfizer's tafamidis for ATTR-CM therapy

The UK’s National Health Service (NHS) has recently started offering Pfizer's tafamidis for treating transthyretin amyloidosis cardiomyopathy (ATTR-CM), a rare heart disease marked by symptoms such as shortness of breath, fatigue, palpitations, fainting, and chest pain. This development follows the National Institute of Health and Care Excellence (NICE) approving the drug for ATTR-CM patients in the UK.

Tafamidis has shown to significantly reduce the risks of hospitalisation and mortality for those suffering from ATTR-CM. Delivered in a once-daily capsule, the drug works by slowing the progression of the disease through the prevention of protein deposits accumulating in the heart. Clinical trials have highlighted its effectiveness, demonstrating a 41% decrease in mortality risk for patients treated with tafamidis compared to those on a placebo.

The NHS's decision to make tafamidis available is supported by interim funding from the Innovative Medicines Fund, ensuring that over 1,000 eligible patients in England can access this treatment. Professor Simon Ray, NHS England’s national clinical director for heart disease, remarked on the significance of this development. He noted that tafamidis represents a groundbreaking treatment option that offers new hope to people living with this progressive condition. Ray emphasized that the availability of this drug aligns with the NHS’s commitment to providing patients with the latest and most effective treatments, thereby significantly improving their quality of life.

Prior to the introduction of tafamidis, treatment options for ATTR-CM were largely focused on symptom management and supportive care, with no effective means to slow the disease's progression. Pfizer UK's country president, Susan Rienow, acknowledged the importance of NICE’s positive decision and the interim funding, describing it as a significant milestone for eligible patients. She expressed satisfaction that tafamidis would now be accessible throughout the UK, reflecting Pfizer's continuing commitment to ensuring patient access to innovative treatments.

In addition to the introduction of tafamidis, the NHS has also announced plans this month to offer Pfizer's Voxelotor (Oxbryta), a new treatment for patients suffering from sickle cell disease. This move further illustrates the NHS’s efforts to provide cutting-edge therapeutic options that address various severe health conditions, improving patient outcomes and quality of life across the country.