Pfizer’s Vyndaqel set to achieve blockbuster status in 2021 with new EU approval for expanded use in ATTR-CM

Following today’s news that Pfizer’s rare disease oral drug Vyndaqel (tafamidis) has gained a new European Medicines Agency (EMA) approval for treating adults with wild-type or hereditary transthyretin amyloidosis with cardiomyopathy (ATTR-CM);

Sarah Elsayed, Pharma Analyst at GlobalData, a leading data and analytics company, offers her view:

“This new approval will dramatically increase the patient pool eligible for Vyndaqel, especially given its outstanding performance in trials. As such, GlobalData forecasts the drug will generate global sales of $3.8bn by 2025, and achieve its long-anticipated blockbuster status by 2021 with global sales of $1.5bn.

“The drug has presented significant results from its Phase III study ATTR-ACT, which shows that when given to ATTR-CM patients over a 30-month duration, Vyndaqel reduced the risk of mortality by 30% compared to placebo and reduced the frequency of cardiovascular-related hospitalization by 32%.

“In patients with ATTR-CM, a blood protein called transthyretin is defective and breaks easily. The broken protein forms a fibrous substance called amyloid that is deposited in the heart walls causing cardiomyopathy and ultimately heart failure. Vyndaqel, a transthyretin stabilizer, acts by stopping the formation of amyloid and slowing down the progression of the disease.

“There are currently no alternative therapies apart from symptomatic management for this rare and fatal condition, thus Vyndaqel's uptake in Europe is expected to surge upon launch, given its transformative results in the cardiac type of ATTR disease, its relatively low side effects, and the huge unmet need in this market.”