VX-659 + ivacaftor + tezacaftor

Unassigned

New Medicines

Cystic fibrosis (CF) in patients heterozygous for F508del and an MF mutation (F/MF) or homozygous for F508del (F/F)

Information

New molecular entity
Vertex
Vertex

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials

Category

Second-generation corrector candidate. Corrector molecules enhance the number of channels of the cystic fibrosis transmembrane conductance regulator (CFTR) protein at the cell surface [2].
Cystic fibrosis (CF) affects approximately 75,000 people in North America, Europe and Australia and is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. There are approximately 2,000 known mutations in the CFTR gene. The median age of death is in the mid-to-late 20s [1].
Cystic fibrosis (CF) in patients heterozygous for F508del and an MF mutation (F/MF) or homozygous for F508del (F/F)
Oral