What is tafamidis and why has its price been so heavily criticised?
A $225,000 annual list price at launch made it the most expensive cardiovascular drug on the market. By 2024 the price watchdog ICER concluded it needed an 85–95% discount to meet standard cost-effectiveness thresholds.
Tafamidis, sold as Vyndaqel and Vyndamax, treats transthyretin amyloid cardiomyopathy (ATTR-CM), a progressive heart disease caused by protein deposits in the heart. When approved in 2019, it carried a $225,000 annual list price, making it the most expensive cardiovascular drug on the market. The lead investigator of the pivotal trial, Dr Mathew Maurer, publicly argued the price was not justified.
By 2024 the list price had risen to nearly $268,000. The drug-pricing watchdog ICER concluded it would need a discount of 85% to 95% to meet standard cost-effectiveness thresholds - implying a fair annual price of roughly $13,600 to $39,000. A public comment filed with the FDA noted the US price was far higher than in Japan or the UK.