How long does it take for Vyndaqel to start working?

It takes at least 6 months for Vyndaqel to start working and it may take up to 18 months for the full effects to be seen, but these persist for at least 30 months. There was significantly less decline in functional capacity (as measured by a 6-minute walk test [6MWT]) and health status (as measured by the Kansas City Cardiomyopathy Questionnaire-Overall Summary [KCCQ-OS]) with Vyndaqel at 6 months than placebo. This remained consistent through month 30 with a 75m difference in the 6MWT and a 14-point difference in the KCCQ-OS score.

The KCCQ-OS score is composed of four domains including total symptoms (symptom frequency and symptom burden), physical limitation, quality of life, and social limitation. The Overall Summary score and domain scores range from 0 to 100, with higher scores representing better health status. All four domains favored pooled Vyndaqel compared to placebo at month 30.

It may take at least 18 months before Vyndaqel makes a difference in all-cause mortality compared to placebo, but this difference persists and trials have reported a survival rate of 71% in those taking Vyndaqel compared to 57% taking placebo. Those receiving Vyndaqel had 32% fewer hospitalizations due to heart problems, also statistically significant.

Vyndaqel (tafamidis meglumine) is a prescription medication used for the treatment of cardiomyopathy caused by transthyretin amyloidosis (ATTR-CM). ATTR-CM is a rare disease caused by the buildup of a protein called amyloid, which is made of transthyretin, in the main pumping chamber of the heart. This causes the heart to become stiff and makes it hard for it to pump blood around the body, which leads to cardiomyopathy and progressive heart failure. Vyndaqel is a transthyretin stabilizer that works by binding to transthyretin. This stabilizes the tetramer of the transthyretin transport protein and slows the formation of amyloid.