FDA Approves First Therapies for ATTR-CM

The US Food and Drug Administration (FDA) has approved a pair of capsule therapies for the treatment of cardiomyopathy caused by transthyretin mediated amyloidosis (ATTR-CM) in adult patients.

Tafamidis (Vyndamax) and tafamidis meglumine (Vyndaqel) are now the first FDA-approved therapies for ATTR-CM. The serious, rare condition is caused by the buildup of abnormal deposits of amyloid in the organ and tissues which interfere with their normal functioning. Most frequently, these deposits occur in the heart and peripheral nervous system. Patients can suffer from shortness of breath, fatigue, heart failure, loss of consciousness, abnormal heart rhythms, and even death if left untreated in the heart.

Among patients affected in the peripheral nervous system, patients can experience a loss of sensation or mobility in the extremities. The 2 approved therapies were shown to reduce more severe patient outcomes while remaining safe treatment options themselves.

A clinical trial randomized 441 patients with ATTR-CM to receive either tafamidis or placebo. After 30 months, treated patients reported a greater survival rate than those administered placebo, and reported a reduced rate of hospitalizations from cardiovascular problems.

Though the patient population was considered small for a cardiovascular condition, no drug-associated side effects were identified among treated patients. The FDA advises that tafamidis may cause fetal harm when administered to pregnant women, and that female patients should discuss pregnancy planning and prevention with their physician.

Recommended doses will differ among the 2 approved therapies, despite their sharing the same active moiety. The drugs, granted to Pfizer subsidiary FoldRx, were previously granted Orphan Drug designation, while tafamidis meglumine also received Fast Track, Priority Review, and Breakthrough Therapy designations.

Both treatments will be immediately impactful for the at-need patient population.

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