Vyndaqel (tafamidis): Amyloidosis treatment approved by the FDA
Vyndaqel (tafamidis), manufactured by Pfizer, was approved by the US Food and Drug Administration (FDA) for the treatment of heart disease caused by transthyretin mediated amyloidosis in adults.
The FDA's marketing approval happened on 3 May 2019 following a clinical trial where the patient group taking the medicine showed that "the survival rate was higher in the Vyndaqel group than in the placebo group" and was also "shown to reduce the number of hospitalizations for cardiovascular problems." Although the number of participants was small, 441, there were no drug related side effects. It was also granted Fast Track, Priority Review, Breakthrough Therapy and Orphan Drug designations in the US1.
Potentially fatal, amyloidosis is caused when abnormal proteins called amyloids accumulate in the organs and tissues. These deposits happen most frequently in the heart and peripheral nervous system. The resulting heart issues can lead to fatigue, abnormal heart rhythm, heart failure, shortness of breath, loss of consciousness and even death.
Vyndaqel (tafamidis) can be harmful to foetuses and treatment should be discussed with a doctor if pregnant or planning to become pregnant.
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