The European Commission (EC) has approved Vyndaqel (tafamidis) for adults with wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM).

The approval of the Pfizer drug mean that it is the first and only treatment approved in the EU for patients with ATTR-CM, as prior to the decision, treatment options for ATTR-CM were restricted to symptom management or heart and liver transplants.

The commission says that the decision was based on results from the Phase III ATTR-ACT study, the first and only completed global, double-blind, randomised, placebo-controlled clinical trial to investigate a pharmacologic therapy for the treatment of ATTR-CM.

In the study, the drug demonstrated a significant reduction in the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalisations compared to placebo.

The approval "represents a big step forward and reflects Pfizer’s commitment to supporting people living with Rare Disease,” said Owen Marks, head of rare disease, Pfizer UK.

He continued, “We are working with UK authorities to secure access and make the treatment available.”

Professor Zaheer Yousef, consultant cardiologist at University Hospital Wales and Cardiff University also commented: “Patients with cardiac amyloid often have a protracted journey with frequent hospital visits, investigations, and lengthy hospitalisations before a diagnosis is reached. Whilst the emergence of new powerful treatments for this otherwise difficult to treat condition are welcomed, initiatives to facilitate early diagnosis should be supported if patients are to gain the benefits of these treatments.”

ATTR-CM is a rare, under-diagnosed and life-threatening disease characterised by the buildup of abnormal deposits of misfolded protein called amyloid in the heart and is defined by restrictive cardiomyopathy and progressive heart failure. Once diagnosed, the median life expectancy in patients with ATTR-CM, dependent on sub-type, is approximately two to 3.5 years.