FDA OKs Pfizer’s tafamidis to treat cardiac amyloidosis

The FDA has cleared two new drugs, tafamidis and tafamidis meglumine, for the treatment of cardiomyopathy caused by a rare disorder known as transthyretin-mediated amyloidosis (ATTR-CM).

Pfizer’s Vyndamax (tafamidis) and Vyndaqel (tafamidis meglumine) were approved May 3, according to a statement, and are the first-ever FDA-approved treatments for ATTR-CM. The condition is triggered by the abnormal buildup of amyloid proteins in the body’s organs and tissues, primarily in the heart and peripheral nervous system.

“Transthyretin-mediated amyloidosis is a rare, debilitating and often fatal disease,” Norman Stockbridge, MD, PhD, of the FDA’s Center for Drug Evaluation and Research, said in the statement. “The treatments we’re approving today are an important advancement in the treatment of the cardiomyopathy caused by transthyretin-mediated amyloidosis.”

When amyloids accumulate in the heart, affected individuals might experience heart failure, shortness of breath, fatigue, arrhythmias, loss of consciousness and death. When they infiltrate the peripheral nervous system, it can result in a loss of sensation, pain or immobility in the limbs.

According to the FDA, Vyndamax and Vyndaqel were proven safe and effective in a small-scale clinical trial of 441 patients randomized to receive Vyndaqel or a placebo. While Vyndaqel and Vyndamax have the same active moiety—tafamidis—they aren’t substitutable on a milligram-to-milligram basis.

In the trial, patients in the Vyndaqel group saw higher survival rates at 30 months than patients in the placebo group. The drug also minimized heart-related hospitalizations.

The FDA granted Vyndaqel fast track, priority review and breakthrough therapy designations, and both Vyndaqel and Vyndamax received orphan drug designation, which aids in the development of drugs for rare diseases. Approval was ultimately granted to FoldRx, a subsidiary of Pfizer.