Drug intelligence / Profile preview

Symdeko + Trikafta

Development stage
Unknown
Lead developer
Vertex
Modality
Small Molecules
Administration
Oral
01

Overview

Symdeko + Trikafta is a combination therapy consisting of two branded cystic fibrosis transmembrane conductance regulator (CFTR) modulators, each with distinct active ingredients.\n**Symdeko** contains tezacaftor and ivacaftor. Tezacaftor acts as a CFTR corrector, improving the processing and trafficking of the CFTR protein to increase its presence at the cell surface, while ivacaftor is a potentiator that increases chloride transport by enhancing the gating activity of CFTR channels[5][3].\n**Trikafta** is a triple combination therapy containing elexacaftor, tezacaftor, and ivacaftor. Elexacaftor and tezacaftor are both correctors that help more functional CFTR protein reach the cell surface; ivacaftor potentiates this protein’s function once it arrives[1][4]. Both drugs are indicated for treating cystic fibrosis in patients with specific mutations in the CFTR gene—primarily those with at least one F508del mutation or other responsive mutations[1][3][5]. The therapies work synergistically to address different defects caused by various mutations in the CFTR gene.

Other names
tezacaftor/ivacaftor + elexacaftor/tezacaftor/ivacaftor
02

Targets

CFTR (Cystic fibrosis transmembrane conductance regulator)

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