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

Discontinued
Discontinued
Discontinued
Jan 20 · Not listed in Vertex pipeline. As an application for the triple combination of VX-445+tezacaftor+ivacaftor has been filed and approved in the US (rather than for the VX-659 combination), it is assumed development of this combination has been discontinued [7].
Feb 19 · In its latest annual report, Vertex states that in Q4 2018, it reported positive PIII data for VX-659+tezacaftor+ivacaftor. In Q4 19, it expects to report data from PIII trials evaluating the triple combination of VX-445+tezacaftor+ivacaftor and then select the better of the two regimens for potential regulatory approval. Vertex expects to file in the US no later than mid-2019 [9].

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

Evidence based evaluations