Pfizer won approval for two formulations of tafamidis-based drugs, including a first for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM).
The U.S. Food and Drug Administration (FDA) approved the new drug known as Vyndaqel (tafamidis meglumine) for ATTR-CM, which makes this the first drug in the U.S. approved to treat this rare heart disease. In addition to Vyndaqel, the FDA also approved another formulation of tafamidis. The FDA approved also Vyndamax (tafamidis). The two drugs are approved for ATTR-CM in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization
Approval of Vyndaqel was based on Phase III results that showed the therapy reduced the risk of death in patients by 30 percent. In addition to the reduced risk of death, Pfizer said use of tafamidis also reduced the rate of cardiovascular-related hospitalizations by 32 percent for this patient population, compared to placebo.
Paul Levesque, global president of rare disease at Pfizer, said the approvals of Vyndaqel and Vyndamax deliver on the company’s promise to develop life-changing medications. The two tafamidis drugs do that for patients with ATTR-CM, he said.
“This milestone is a game changer for patients, who until today had no approved medicines for this rare, debilitating and fatal disease. We will continue to focus efforts on working with the physician community to increase awareness and ultimately detection and diagnosis of this disease,” Levesque said in a statement.
According to Reuters, Pfizer has set a list price of $225,000 a year for the medicine. Analysts have already pegged the drug as a potential blockbuster, with annual sales expected to exceed $1 billion in 2024, Reuters said.
Pfizer’s price for the ATTR-CM drugs comes in about $200,000 lower than treatments for hereditary TTR amyloidosis, which also caused by a buildup of transthyretin protein in the body. Drugs developed by Alnylam Pharmaceuticals and Ionis Pharmaceuticals for that disease have a price of about $450,000 a year, Reuters noted.