Thursday, September 19, 2019

Thursday, September 19, 2019

New treatments for transthyretin mediated amyloidosis approved

Two new medicines, Vyndaqel and Vyndamax, were approved for adults by the Food and Drug Administration earlier this month. They will help to treat heart disease caused by transthyretin mediated amyloidosis (ATTR-CM).

“Transthyretin-mediated amyloidosis is a rare, debilitating and often fatal disease,” said Norman Stockbridge, 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.”

ATTR most often affects the heart and peripheral nervous system when the protein amyloid builds up in the organs. This can cause symptoms which range from shortness of breath to loss of consciousness, pain, immobility and death.

In clinical trials, the medicine was shown to help the group of patients who received the drug better than the group who received a placebo. After 30 months, Vyndaqel reduced the number of hospitalizations and increased the survival rate.

There are no known drug-associated side effects, but women should consult their doctor before taking the medicine if they are pregnant or plan to become pregnant.

In addition to the approval of the two drugs, the FDA granted Fast Track, Priority Review, and Breakthrough Therapy designations for Vyndaqel and both medicines received Orphan Drug designations. These designations will help with future development of drugs for rare diseases.

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US Food and Drug Administration

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