Pfizer's Rare Disease Drug Tafamidis Soft Capsules Approved in China for ATTR-CM

By Feng Qiu

On October 9, the National Medical Products Administration (NMPA) approved Pfizer’s marketing application for tafamidis meglumine soft capsules in China, for the treatment of adult patients with wild-type or hereditary transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) to reduce cardiovascular mortality and cardiovascular-related hospitalizations.

ATTR-CM is a rare, fatal disease caused by the instability of transthyretin, a transport protein. Transthyretin consists of four identical subunits (a tetramer). When unstable transthyretin tetramers dissociate, they lead to the formation of misfolded proteins that deposit as amyloid fibrils in the heart, causing myocardial stiffness and ultimately resulting in heart failure.

In May 2019, Pfizer’s Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) received FDA approval for marketing. These are two oral formulations of tafamidis, a transthyretin stabilizer that selectively binds to transthyretin and stabilizes its tetrameric structure. On February 5, 2020, Pfizer’s tafamidis meglumine soft capsules (Vyndaqel) became the first to receive approval from the National Medical Products Administration (NMPA) in China for the treatment of adult patients with stage I symptomatic transthyretin amyloidosis with polyneuropathy (ATTR-PN), aiming to delay the progression of peripheral neurological impairment.