Pfizer's Tafamidis Soft Capsules Nearing Approval in China for ATTR-CM Treatment

By Baihuawen

On September 21, Pfizer’s marketing application for tafamidis soft capsules in China entered the “under review” stage, with expectations of formal approval in the near future for the treatment of adults with wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM) to reduce cardiovascular mortality and cardiovascular-related hospitalizations.

ATTR-CM is a rare, fatal disease caused by the instability of the transport protein transthyretin. 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, the FDA approved Pfizer’s Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) for marketing. These are two oral formulations of tafamidis, a transthyretin stabilizer that selectively binds to transthyretin, stabilizes its tetrameric structure, and slows the formation of amyloid deposits responsible for ATTR-CM.

Notably, Vyndaqel (tafamidis meglumine) was approved in China in February 2020 for the treatment of stage I symptomatic adult patients with transthyretin amyloid polyneuropathy (ATTR-PN) to delay peripheral neurological impairment.