Tezacaftor is a

drug A drug is any chemical substance that causes a change in an organism's physiology or psychology when consumed. Drugs are typically distinguished from food and substances that provide nutritional support. Consumption of drugs can be via insuffla ...

used for the treatment of

cystic fibrosis Cystic fibrosis (CF) is a rare genetic disorder that affects mostly the lungs, but also the pancreas, liver, kidneys, and intestine. Long-term issues include difficulty breathing and coughing up mucus as a result of frequent lung infections. O ...

(CF) in people six years and older, who have a F508del mutation, the most common type of mutation in the CFTR 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 Elexacaftor is a medication that acts as cystic fibrosis transmembrane conductance regulator (CFTR) corrector. It is available in a single pill with ivacaftor and tezacaftor; the fixed-dose combination, elexacaftor/tezacaftor/ivacaftor (brand na ...

, tezacaftor, and

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 chl ...

. In 2019, the U.S. Food and Drug Administration (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 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

, specifically with the homozygous for Phe508del mutation. The EVOLVE trial is a

phase 3 Phase 3, Phase III or Phase Three may refer to: Media * Marvel Cinematic Universe: Phase Three, eleven American superhero films from 2016–2019 * '' Phase 3: Thrones and Dominions'', a 1995 album by Earth * ''Phase III'' (album), a 1972 album ...

, double-blinded, multicenter, randomized, placebo-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 (FDA) granted the application for tezacaftor and ivacaftor combination therapy

orphan drug An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of ...

designation and priority review, and granted the approval of Symdeko to

Vertex Pharmaceuticals Incorporated Vertex Pharmaceuticals is an American biopharmaceutical company based in Boston, Massachusetts. It was one of the first biotech firms to use an explicit strategy of rational drug design rather than combinatorial chemistry. It maintains headqua ...

References

External links

* * Benzodioxoles Cyclopropanes Indoles Organofluorides Orphan drugs {{respiratory-system-drug-stub