Transthyretin amyloid cardiomyopathy

New treatments for rare disease ATTR-CM approved

The U.S. Food and Drug Administration (FDA) approved Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules for the treatment of the heart disease (cardiomyopathy) caused by transthyretin mediated amyloidosis (ATTR-CM) in adults.

These are the first FDA-approved treatments for 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, M.D., Ph.D., director of the Division of Cardiovascular and Renal Drugs in the FDA’s Center 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.”

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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. Heart involvement can result in shortness of breath, fatigue, heart failure, loss of consciousness, abnormal heart rhythms and death. Involvement of the peripheral nervous system can result in a loss of sensation, pain, or immobility in the arms, legs, hands and feet. Amyloid deposits can also affect the kidneys, eyes, gastrointestinal tract and central nervous system.

The efficacy of Vyndaqel and Vyndamax in treating ATTR-CM was shown in a clinical trial of 441 patients randomized 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 hospitalizations for cardiovascular problems. The number of patients in clinical studies was small, but no drug-associated side effects have been identified. 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 designation, which provides incentives to assist and encourage the development of drugs for rare diseases. Approval of Vyndaqel and Vyndamax were granted to FoldRx, a subsidiary of Pfizer.


Source: FDA

06.05.2019

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