Pfizer’s Tafamidis Approved in First for Rare Heart Disease

Pfizer_Editorial_Roman Tiraspolsky

Roman Tiraspolsky /

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.

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Transthyretin amyloid cardiomyopathy is a rare, life-threatening disease characterized by the buildup of abnormal deposits of misfolded protein called amyloid in the heart and is defined by restrictive cardiomyopathy and progressive heart failure. Prior to the new approvals, the only available options included symptom management, and, in rare cases, heart (or heart and liver) transplant. Pfizer estimates there are about 100,000 people in the U.S. with ATTR-CM and only one to two percent of those patients are diagnosed today.

Pfizer’s 441-patient study included those who had the hereditary form of the disease, as well as the “wild-type form,” meaning it can occur as people age. Following the announcement of topline results in May 2018, the U.S. Food and Drug Administration granted the drug Breakthrough Therapy Designation. In 2017 the FDA granted Fast Track designation to Tafamidis for transthyretin cardiomyopathy.

Brenda Cooperstone, chief development officer in Pfizer’s rare disease business, said Vyndaqel and Vyndamax reduce cardiovascular mortality, as well as the frequency of cardiovascular-related hospital stays in patients with wild-type or hereditary forms of this disease. The drugs provide these patients with a chance for more time with their loved ones,” she said.

Pfizer has tried for approval for of Tafamidis before as a treatment for a neurodegenerative disease, but was soundly rejected by the U.S. Food and Drug Administration. Pfizer acquired Tafamidis in 2010 as part of the deal for FoldRx. Tafamidis has been approved in Europe for transthyretin familial amyloid polyneuropathy (TTR-FAP). It is sold there under the Vyndaqel brand name.

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.

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