The FDA has approved tafamidis meglumine and tafamidis (Vyndaquel® and VyndamaxTM, Pfizer Inc.) for the treatment of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. Both drugs are oral formulations of the first-in-class transthyretin stabilizer tafamidis.
The rare and life-threatening ATTR-CM is characterized by the buildup of abnormal deposits of amyloid protein in the heart, causing restrictive cardiomyopathy and progressive heart failure.
The approval was based on data from the pivotal phase 3 Transthyretin Amyloidosis Cardiomyopathy Clinical Trial (ATTR-ACT), the first global, double-blind, randomized, placebo-controlled clinical study to investigate a pharmacological therapy for ATTR-CM treatment.
In the trial, tafamidis meglumine significantly reduced all-cause mortality and frequency of cardiovascular-related hospitalizations compared with placebo, over 30 months. Tafamidis meglumine also showed significant improvement compared with placebo in patients’ functional capacity and health status at six months and continuing through 30 months.
Previously, the sole treatment options were symptom management and, rarely, heart (or heart and liver) transplant. Approximately 100,000 people in the U.S are thought to have ATTR-CM, but only one to two percent of them have been diagnosed. Following diagnosis, the median life expectancy in patients with ATTR-CM is approximately two to 3.5 years.
Source: Pfizer Inc, May 6, 2019