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.”

Recommended article

Photo

Cardiology congress

ESC 2018: At the heart of research

When delegates from around 150 countries converge on Munich for ESC Congress 2018 they will no doubt reflect on what they themselves eat. Yes, nutrition is up for debate, questioning, for example, whether weight loss therapies can also prevent heart attacks and strokes. Results from the CAMELLIA-TIMI 61 trial of 12,000 overweight individuals with established cardiovascular disease or diabetes…

graffiti of stick figure man hanging on to red heart
Source: Unsplash/Nick Fewings

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

More on the subject:
Read all latest stories

Related articles

Photo

Research

First use of vasoprotective antibody in cardiogenic shock

Scientists at the German Centre for Cardiovascular Research (DZHK) have started a study to find out whether a monoclonal antibody restoring vascular integrity is safe and has positive effects on…

Photo

Gender issues

Clinical trial enrolment favours men

Clinical trial enrolment favours men, according to a study presented at Heart Failure 2018 and the World Congress on Acute Heart Failure, a European Society of Cardiology congress.1 The study found…

Photo

Drug development

New research could help address heart problems in diabetics

Research published in Experimental Physiology shows that diabetes-induced changes in heartbeat are primarily regulated by the β1-adrenoceptor. This discovery, once confirmed in humans, may lead to…

Related products