Tezacaftor is a
drug used for the treatment of
cystic fibrosis (CF) in people six years and older, who have a F508del mutation, the most common type of mutation in the
CFTR
Cystic fibrosis transmembrane conductance regulator (CFTR) is a membrane protein and anion channel in vertebrates that is encoded by the ''CFTR'' gene.
Geneticist Lap-Chee Tsui and his team identified the CFTR gene in 1989 as the gene linked wi ...
gene.
It is sold as a fixed-dose combination with
ivacaftor
Ivacaftor is a medication used to treat cystic fibrosis in people with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (primarily the G551D mutation), who account for 4–5% cases of cystic fibrosis. It ...
under the brand name Symdeko.
It was approved by the U.S. FDA in 2018.
The combination of
elexacaftor, tezacaftor, and
ivacaftor
Ivacaftor is a medication used to treat cystic fibrosis in people with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (primarily the G551D mutation), who account for 4–5% cases of cystic fibrosis. It ...
is being sold as
Trikafta
Elexacaftor/tezacaftor/ivacaftor, sold under the brand names Trikafta (US) and Kaftrio (EU), is a fixed-dose combination medication used to treat cystic fibrosis. Elexacaftor/tezacaftor/ivacaftor is composed of a combination of ivacaftor, a c ...
.
In 2019, the U.S.
Food and Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respon ...
(FDA) approved a combination of elexacaftor, tezacaftor, and ivacaftor.
[ ]
Mechanism of action
Tezacaftor acts as a corrector to help the folding and presentation of the
CFTR
Cystic fibrosis transmembrane conductance regulator (CFTR) is a membrane protein and anion channel in vertebrates that is encoded by the ''CFTR'' gene.
Geneticist Lap-Chee Tsui and his team identified the CFTR gene in 1989 as the gene linked wi ...
protein to the cell surface, which improves its function for individuals with a F508del mutation.
Clinical trials
The EVOLVE and EXPAND study findings were published in 2017.
EVOLVE trial
The EVOLVE trial analyzed tezacaftor/ivacaftor in patients with
cystic fibrosis, specifically with the homozygous for Phe508del mutation.
The EVOLVE trial is a
phase 3, double-blinded, multicenter, randomized,
placebo
A placebo ( ) is a substance or treatment which is designed to have no therapeutic value. Common placebos include inert tablets (like sugar pills), inert injections (like saline), sham surgery, and other procedures.
In general, placebos can af ...
-controlled, parallel-group trial, that was which evaluated therapy with a combination of tezacaftor and ivacaftor in patients that are 12 and older.
510 patients were randomized and 509 patients were given either 100 mg of tezacaftor once daily and 150 mg of ivacaftor twice daily or a placebo for 24 weeks.
The combination of drugs was efficacious in patients who had cystic fibrosis with the Phe508del mutation and the adverse effects in both treatment groups were similar.
History
The U.S.
Food and Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respon ...
(FDA) granted the application for tezacaftor and ivacaftor combination therapy
orphan drug designation and
priority review
Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program ...
, and granted the approval of Symdeko to
Vertex Pharmaceuticals Incorporated.
References
External links
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Benzodioxoles
Cyclopropanes
Indoles
Organofluorides
Orphan drugs
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