General Information

Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) are oral transthyretin stabilizers. Vyndamax was developed for patient convenience. Vyndaqel and Vyndamax are not substitutable on a per milligram basis.

Vyndaqel and Vyndamax are specifically indicated for the treatment of the cardiomyopathy of wild type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization.

Vyndaqel and Vyndamax are supplied as capsules for oral administration. The recommended dose is either Vyndaqel 80 mg (four 20-mg tafamidis meglumine capsules) orally once daily or Vyndanmax 61 mg (one 61-mg tafamidis capsule) orally once daily.

Mechanism of Action

Vyndaqel (tafamidis meglumine) is an oral transthyretin stabilizer that selectively bind to transthyretin, stabilizing the tetramer of the transthyretin transport protein and slowing the formation of amyloid that causes ATTR-CM.

Side Effects

In clinical trials, the frequency of adverse events in patients treated with Vyndaqel was similar to placebo, and similar proportions of Vyndaqel-treated patients and placebo-treated patients discontinued the study drug because of an adverse event.

Clinical Trial Results

The FDA approval was based on a multicenter, international, randomized, double-blind, placebo-controlled study in 441 patients with wild type or hereditary ATTR-CM. Patients were randomized in a 1:2:2 ratio to receive Vyndaqel 20 mg (n=88), Vyndaqel  80 mg (administered as four 20-mg Vyndaqel capsules) (n=176), or matching placebo (n=177) once daily for 30 months, in addition to standard of care (e.g., diuretics). Treatment assignment was stratified by the presence or absence of a variant TTR genotype as well as baseline disease severity (NYHA Class). Transplant patients were excluded from this study. The analysis demonstrated a significant reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations in the pooled Vyndaqel 20 mg and 80 mg arms versus placebo. Analysis of the individual components of the primary analysis (all-cause mortality and cardiovascular-related hospitalizations) also demonstrated significant reductions for Vyndaqel versus placebo.