FDA Approves First Treatment For ATTR-CM

On May 3, the U.S. Food and Drug Administration (FDA) approved Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules for the treatment of cardiomyopathy caused by transthyretin mediated amyloidosis (ATTR-CM) in adults, making them the "first FDA-approved treatments for ATTR-CM." ATTR-CM is a rare, progressive and fatal disease that is either hereditary (mutations in the TTR gene) or acquired, and typically presents in patients between 50 years and 80 years.

Findings from the ATTR-ACT trial presented during ESC Congress 2018 and published in the New England Journal of Medicine showed that tafamidis is an effective and safe therapy for treating patients with ATTR-CM.

According to the FDA's press release, "The number of patients in clinical studies was small, but no drug-associated side effects have been identified. Tafamidis may cause fetal harm when administered to a pregnant woman."

Read more on FDA's website.

Clinical Topics: Heart Failure and Cardiomyopathies

Keywords: ACC Advocacy, Prealbumin, Capsules, United States Food and Drug Administration, Benzoxazoles, Amyloidosis, Cardiomyopathies, Mutation

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