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Tezacaftor · Alyftrek, Symdeko

Small molecule · Modulator of CFTR · developed by Vertex Pharmaceuticals

Approved FDA

VRTX Developer

3 Registered trials

What is Tezacaftor?

Tezacaftor is a CFTR modulator that binds to the CFTR protein and facilitates cellular processing and trafficking of select mutant CFTR forms, including F508del-CFTR, to increase CFTR protein at the cell surface. It is used in combination with other CFTR modulators for treating cystic fibrosis in patients 6 years and older with responsive CFTR variants.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as: Alyftrek, Symdeko, Trikafta, VX-121, TEZ, VX-661
Drug type: Small molecule
Targets: CFTR
Mechanism: Modulator of CFTR
Pathways: CFTR-mediated chloride transport
Therapy area: Respiratory
Developer: Vertex Pharmaceuticals Incorporated (VRTX)
FDA status: Approved as Alyftrek, Symdeko, Trikafta, NDA218730 · FDA label
UNII: 8RW88Y506K

✅ FDA-approved uses

Cystic fibrosis (Symdeko)
Cystic fibrosis (Trikafta)
Cystic fibrosis (Alyftrek)

From the indications section of the FDA label (version of Aug 21, 2026). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT05422222	Phase 3	Cystic Fibrosis	Active not recruiting	VRTX
NCT07437105	Phase 1	Cystic Fibrosis	Recruiting	VRTX
NCT07283770	Phase 1	Cystic Fibrosis	Recruiting	VRTX

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Cystic Fibrosis

Other drugs acting on CFTR

Deutivacaftor FDA approved Elexacaftor FDA approved Ivacaftor FDA approved Vanzacaftor FDA approved

More from Vertex Pharmaceuticals

Deutivacaftor FDA approved Elexacaftor FDA approved Exagamglogene Autotemcel FDA approved Ivacaftor FDA approved Suzetrigine FDA approved Vanzacaftor FDA approved Inaxaplin Phase 2/3 Povetacicept Phase 2/3 VX-017 Phase 3 VX-118 Phase 1 VX-264 Phase 1/2 VX-272 Phase 1

Sources

Updated Oct 3, 2026. Something wrong or missing? Report it on the VRTX page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z