Tafamidis for ATTR-CM in US

July 5, 2019 0 By FM

Pfizer said tafamidis has been granted approval by the US FDA to treat transthyretin amyloid cardiomyopathy (ATTR-CM).

ATTR-CM is a rare, life-threatening disease characterized by the build-up of abnormal deposits of a misfolded protein called amyloid in the heart and is defined by restrictive cardiomyopathy and progressive heart failure.

Previously, there were no medicines approved to treat ATTR-CM.

The FDA 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 pharmacological therapy for the treatment of this disease.

In ATTR-ACT, tafamidis significantly reduced the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo over a 30-month period.

Additionally, individual components of the primary analysis demonstrated a relative reduction in the risk of all-cause mortality and frequency of cardiovascular-related hospitalization of 30% and 32%.

An oral agent, tafamidis is a 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.