Pfizer's Vyndaqel, Vyndamax win US nod for rare disease

The US Food and Drug Administration (FDA) has approved Pfizer's Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) for use in patients with transthyretin amyloid cardiomyopathy, a rare and fatal disease.

The indication is for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular related deaths and hospitalisation, making them the only two oral formulations of the first-in-class transthyretin stabilizer tafamidis, and the first and only medicines approved by the FDA to treat ATTR-CM.

The approval is based on a clinical trial which showed that after an average of 30 months, the survival rate was higher in the Vyndaqel group than in the placebo group. Vyndaqel was also shown to reduce the number of hospitalizations for cardiovascular problems.

The approvals of "Vyndaqel and Vyndamax are a testament to the significant research and development investment in our innovative cardiovascular outcomes trial, ATTR-ACT. We are proud to bring these medicines to ATTR-CM patients who are in dire need of treatment," said Brenda Cooperstone, senior vice president and chief development officer, rare disease, Pfizer Global Product Development.

"Vyndaqel and Vyndamax reduce cardiovascular mortality and the frequency of cardiovascular-related hospital stays in patients with wild-type or hereditary forms of this rare disease, giving them a chance for more time with their loved ones."

ATTR is caused by the buildup of abnormal deposits of specific proteins known as amyloid in the body's organs and tissues, interfering with their normal functioning. These protein deposits most frequently occur in the heart and the peripheral nervous system.

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