drug

(deutivacaftor + tezacaftor + vanzacaftor calcium)

Marketedupdated 2mo ago

by $VERX Vertex, Inc.

Approved

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Small molecule drug with a maximum clinical stage of Approval, with an approval for cystic fibrosis.

indications

investigational

Cystic Fibrosis

all catalysts(4)

approval · 4

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brand

Alyftrek

generic

(deutivacaftor + tezacaftor + vanzacaftor calcium)

MOA

12.1 Mechanism of Action Vanzacaftor and tezacaftor bind to different sites on the CFTR protein and have an additive effect in facilitating the cellular processing and trafficking of select mutant forms of CFTR (including F508del- CFTR) to increase the amount of CFTR protein delivered to the cell surface compared to either molecule alone. Deutivacaftor potentiates the channel open probability (or gating) of the CFTR protein at the cell surface. The combined effect of vanzacaftor, tezacaftor and deutivacaftor is increased quantity and function of CFTR at the cell surface, resulting in increased CFTR activity as measured both by CFTR-mediated chloride transport in vitro and by sweat chloride in patients with CF. CFTR Chloride Transport Assay in Fischer Rat Thyroid Cells Expressing Mutant CFTR Protein Effects of vanzacaftor/tezacaftor/deutivacaftor on chloride transport for mutant CFTR protein was determined in Ussing chamber electrophysiology studies using a panel of Fischer Rat Thyroid (FRT) cell lines stably expressing CFTR protein from individual variants. Vanzacaftor/tezacaftor/deutivacaftor increased chloride transport in FRT cells expressing select CFTR variants, as identified in Table 5. The threshold that the treatment-induced increase in chloride transport must exceed for the mutant CFTR protein to be considered responsive is ≥10% of normal over baseline. This threshold was used because it is expected to predict clinical benefit. For individual variants, the magnitude of the net change over baseline in CFTR-mediated chloride transport in vitro is not correlated with the magnitude of clinical response. CFTR Chloride Transport Assay in Human Bronchial Epithelial Cells Expressing Mutant CFTR Protein Homozygous and heterozygous N1303K- Human Bronchial Epithelial (HBE) cells showed greater chloride transport in the presence of vanzacaftor/tezacaftor/deutivacaftor than F508del/F508del- HBE cells treated with tezacaftor/ivacaftor which has shown clinical benefit in

indication

Cystic Fibrosis

phase

marketed

(deutivacaftor + tezacaftor + vanzacaftor calcium)

indications

all catalysts(4)

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patents (61)

FDA exclusivities (2)

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