FDA Drug Approvals, Cardiology — 2019 Midyear Review

Mary L Windle, PharmD


August 08, 2019

Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis)

Tafamidis meglumine and tafamidis are indicated for cardiomyopathy caused by transthyretin-mediated amyloidosis (ATTR-CM) in adults. These are the first FDA-approved treatments for ATTR-CM. Although each product has the same active moiety, tafamidis, they are not substitutable on a milligram-to milligram basis, and their recommended doses differ.

Accumulation of amyloid fibrils composed of misfolded transthyretin protein in the heart leads to transthyretin amyloid cardiomyopathy and heart failure. Drugs that stabilize the amyloidogenic process may slow disease progression. Tafamidis is a selective stabilizer of TTR. Tafamidis binds to TTR at the thyroxine binding sites, stabilizing the tetramer and slowing dissociation into monomers, the rate-limiting step in the amyloidogenic process.

Efficacy of tafamidis was shown in a clinical trial of 441 patients randomized to receive tafamidis meglumine or a placebo. All-cause mortality and rates of cardiovascular-related hospitalizations were lower among the 264 patients who received tafamidis than among the 177 patients who received placebo (P<.001). After an average of 30 months, tafamidis was also associated with a lower rate of decline in distance for the 6-minute walk test (P<.001) and a lower rate of decline in the Kansas City Cardiomyopathy Questionnaire-Overall Summary (KCCQ-OS) score (P<.001).[1]

Other notable cardiology approval

Praluent (alirocumab) gained FDA approval to reduce risk of myocardial infarction, stroke, and unstable angina requiring hospitalization in adults with cardiovascular disease.


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