How does Vyndaqel work for ATTR-CM?

Vyndaqel works in ATTR-CM (cardiomyopathy of transthyretin-mediated amyloidosis) by binding to and stabilizing the transthyretin (TTR) transport protein. This slows down the production of amyloid deposits in the heart that lead to ATTR-CM.

Vyndaqel is a prescription medicine for ATTR-CM that helps to reduce hospitalization and death related to heart problems. It is taken by mouth as four 20-mg capsules once a day.

ATTR-CM is a very rare disease due to a build-up of the amyloid protein called transthyretin in the heart. This leads to cardiomyopathy and eventual heart failure because the heart cannot function and pump blood adequately.

Transthyretin (TTR) is a protein primarily made in the liver. TTR carries thyroxine (a thyroid hormone) and retinol (vitamin A) throughout the body. Transthyretin-mediated amyloidosis (ATTR) results in misfolded, abnormal deposits of the TTR protein, which build-up in the body and can cause organ damage. A build-up of TTR in the heart can lead to heart failure. There is no cure for ATTR and it may remain undiagnosed in many patients.

Vyndaqel (tafamidis meglumine) is used in adults with hereditary or wild type ATTR to treat cardiomyopathy (damage of the heart muscle) caused by a build-up of transthyretin (TTR).

• People with hereditary ATTR amyloidosis have a DNA mutation that can be passed from one generation to the next.
• People with wild-type ATTR amyloidosis do not have abnormal DNA and cannot pass on the condition.

Amyloidosis can also affect other organs like the kidneys, liver, spleen, nervous system, stomach or intestines, but Vyndaqel is only used for the heart.