The US FDA has recently granted priority review and breakthrough therapy designations for treating two Tafamidis drug formulations of different recommended doses for the treatment of a rare heart disease known as Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Both tafamidis and tafamidis meglumine have same active moiety but their recommended doses differ. ATTR-CM is a rare, and fatal disease-causing abnormal deposits of specialized amyloid proteins in the organs and tissues.
These proteins hinder the normal functioning of the heart and peripheral nervous system as their deposits are found in those locations. Associated risks when the heart is involved: Shortness of breath Fatigue Heart failure Loss of consciousness Abnormal heart rhythms Death Associated risks when the peripheral nervous system is involved: Loss of sensation Pain Immobility in the arms, legs, hands, and feet The…