FDA approves novel treatments for heart disease

The US Food and Drug Administration (FDA) approved Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules for this treatment, which are the first FDA-approved treatments for transthyretin mediated amyloidosis (ATTR-CM).

Vyndaqel and Vyndamax have the same active moiety, tafamidis, but they are not substitutable on a milligram to milligram basis and their recommended doses differ.

“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 Centre for Drug Evaluation and Research. “The treatments we’re approving today are an important advancement in the treatment of the cardiomyopathy caused by transthyretin-mediated amyloidosis.”

The efficacy of Vyndaqel and Vyndamax in treating ATTR-CM was shown in a clinical trial of 441 patients randomised to receive Vyndaqel or a placebo. 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 hospitalisations for cardiovascular problems.

The number of patients in clinical studies was small, but no drug-associated side effects have been identified. However, tafamidis may cause fetal harm when administered to a pregnant woman. Women taking Vyndaqel or Vyndamax should discuss pregnancy planning and prevention with their health care professional.

The FDA granted Vyndaqel Fast Track, Priority Review and Breakthrough Therapy designations. Vyndaqel and Vyndamax each received Orphan Drug designations, which provides incentives to assist and encourage the development of drugs for rare diseases.