The US Food and Drug Administration (FDA) has granted breakthrough therapy designation to Pfizer’s tafamidis to treat patients suffering from transthyretin cardiomyopathy, a rare condition characterised by progressive heart failure.
Tafamidis is an investigational drug and secured orphan drug designation for transthyretin cardiomyopathy in 2012 in the US and the European Union (EU).
In May last year, the product received the FDA fast track status, while the Japanese Ministry of Labor Health and Welfare granted it SAKIGAKE designation in March this year for the same indication.
Pfizer Global Product Development, Rare Disease senior vice-president Brenda Cooperstone said: “This designation is an important step forward in the path to bringing a potential new treatment option to those with transthyretin cardiomyopathy, a rare, fatal disease.We look forward to working with the FDA through this expedited process to fulfil an unmet patient need.”
The FDA based its approval on the top-line results obtained from the Phase III Transthyretin Cardiomyopathy (ATTR-ACT) clinical trial.
Results revealed that tafamidis led to a statistically significant decrease in the combination of all-cause mortality and the number of cardiovascular-related hospitalisations.
Currently, transthyretin cardiomyopathy lacks specific, approved pharmacological treatments. The average life expectancy for people suffering from the disease is reported to be around three to five years after diagnosis.