The Ministry of Food and Drug Safety has approved Pfizer's Vyndamax (ingredient: tafamidis), a treatment for wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM), for reduced cardiovascular mortality and associated hospitalization in adult patients.

Vyndamax is the first and only approved therapy for treating wild-type or hereditary ATTR-CM in adult patients by taking one tab a day.

Pfizer Korea has recently won a regulatory nod for its hereditary transthyretin amyloid drug Vynadamax (ingredient: tafamidis). (Pfizer)

ATTR-CM is a rare and fatal debilitating disease that causes limited cardiomyopathy due to unstable transthyretin, a transporting protein that naturally circulates in the blood, separating into misfolded units and accumulating in the heart.

The ministry approved the drug based on the multi-center, international, double-blinded, and placebo-controlled phase 3 trial, ATTR-ACT, of 441 ATTR-CM patients.

In the ATTR-ACT study, 441 patients were randomly assigned in a 2:1:2 ratio to receive 80 mg tafamidis, 20 mg tafamidis, or placebo, respectively. The researchers hierarchically tested the frequency of deaths caused by deaths and cardiovascular-related hospitalization for all causes as the primary endpoint in the study.

The secondary endpoint of the study was the six-minute walk test of 30 months from the baseline and the changes in the Kansas City Cardiomyopathy Questionnaire-Overall Summary score, indicating better health status with higher scores.

As a result of the study, the tafamidis cohort of 264 patients had a statistically lower risk of death of all causes and hospitalization rates linked to cardiovascular than those of the placebo group of 177 patients.

At the time of 30 months from the baseline, patients showed improvement in their functional athletic ability in the six-minute walk test and reduction in the Kansas City Cardiomyopathy Questionnaire score, which evaluates the patient's quality of life. The first significant difference compared to the placebo group was observed in the sixth month of the study.

"We are pleased to provide a therapeutic option for ATTR-CM, which had no way of treating the disease other than the heart or liver transplant, with the recent regulatory approval of our innovative drug Vyndamax," said Cho Yeon-jin, head of the Rare Disease Department of Pfizer Korea.

Vyndamax was approved as a therapy for ATTR-CM in the U.S., followed by Canada and Europe in 2020, and also had been designated as an orphan drug in the U.S. and Europe.

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