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Vanzacaftor · Alyftrek, VX-121

Small molecule · Modulator of CFTR · developed by Vertex Pharmaceuticals

Approved FDA

VRTX Developer

1 Registered trials

What is Vanzacaftor?

Vanzacaftor is a component of ALYFTREK, a fixed-dose combination with tezacaftor and deutivacaftor. Vanzacaftor and tezacaftor bind different sites on CFTR to enhance its cellular processing and trafficking, while deutivacaftor potentiates CFTR channel gating. Together they increase CFTR quantity and function at the cell surface, enhancing chloride transport in cystic fibrosis patients 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, VX-121, VNZ/TEZ/D-IVA
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, NDA218730 · FDA label
UNII: COM1POP492

✅ FDA-approved uses

Cystic fibrosis

From the indications section of the FDA label (version of Aug 26, 2026). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT05844449	Phase 3	Cystic Fibrosis	Enrolling by invitation	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 Tezacaftor FDA approved

More from Vertex Pharmaceuticals

Deutivacaftor FDA approved Elexacaftor FDA approved Exagamglogene Autotemcel FDA approved Ivacaftor FDA approved Suzetrigine FDA approved Tezacaftor 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