Tezacaftor

Overview

Tezacaftor is a drug of the cystic fibrosis transmembrane conductance regulator (CFTR) potentiator class. It was developed by Vertex Pharmaceuticals and FDA approved in combination with ivacaftor to manage cystic fibrosis. This drug was approved by the FDA on February 12, 2018. Cystic Fibrosis is an autosomal recessive disorder caused by one of several different mutations in the gene for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, an ion channel involved in the transport of chloride and sodium ions across cell membranes. CFTR is active in epithelial cells of organs such as of the lungs, pancreas, liver, digestive system, and reproductive tract. Alterations in the CFTR gene result in altered production, misfolding, or function of the protein and consequently abnormal fluid and ion transport across cell membranes. As a result, CF patients produce thick, sticky mucus that clogs the ducts of organs where it is produced making patients more susceptible to complications such as infections, lung damage, pancreatic insufficiency, and malnutrition.

Indication

Tezacaftor is combined with ivacaftor in one product for the treatment of cystic fibrosis (CF) in patients aged 12 years or older with two copies of the F508del gene mutation or at least one mutation in the CFTR gene that is responsive to this drug. Tezacaftor, when used in combination with ivacaftor and elexacaftor in the product Trikafta, is also indicated for the treatment of CF in patients 12 years of age and older that have at least one F508del mutation in the CFTR gene.

Associated Conditions

Cystic Fibrosis (CF)

Clinical Trials

Title	Posted	Study ID	Phase	Status	Sponsor
Evaluation of VX-828 in Healthy Participants and in Participants With Cystic Fibrosis	2023/12/04	NCT06154447	Phase 1	Recruiting	Vertex Pharmaceuticals Incorporated
Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations	2018/08/09	NCT03624101	Phase 4	Terminated	University of Alabama at Birmingham
Personalized Theratyping Trial	2018/07/16	NCT03587961	Early Phase 1	Recruiting	University of Alabama at Birmingham
A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis	2017/01/24	NCT03029455	Phase 1	Completed	Vertex Pharmaceuticals Incorporated
Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion	2014/02/25	NCT02070744	Phase 2	Completed	Vertex Pharmaceuticals Incorporated
An Open-Label, Phase 1 Study in Healthy Adult Subjects to Examine the Effects of Multiple-Dose Ciprofloxacin on Ivacaftor and VX-661 in Combination With Ivacaftor	2013/12/19	NCT02015507	Phase 1	Completed	Vertex Pharmaceuticals Incorporated
Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation	2012/02/13	NCT01531673	Phase 2	Completed	Vertex Pharmaceuticals Incorporated

FDA Drug Approvals

Approved Product	Manufacturer	NDC Code	Route	Strength	Effective Date
No FDA approvals found for this drug.

EMA Drug Approvals

Approved Product	Authorization Holder	Status	Issued Date
Kaftrio	Vertex Pharmaceuticals (Ireland) Limited,Unit 49, Block 5,Northwood Court,Northwood Crescent,Dublin 9,D09 T665,Ireland	Authorised	8/21/2020
Kaftrio	Vertex Pharmaceuticals (Ireland) Limited,Unit 49, Block 5,Northwood Court,Northwood Crescent,Dublin 9,D09 T665,Ireland	Authorised	8/21/2020
Symkevi	Vertex Pharmaceuticals (Ireland) Limited,Unit 49, Block 5,Northwood Court,Northwood Crescent,Dublin 9,D09 T665,Ireland	Authorised	10/31/2018

HSA Drug Approvals

Approved Product	Manufacturer	Approval Number	Dosage Form	Strength	Approval Date
No HSA approvals found for this drug.

NMPA Drug Approvals

Approved Product	Company	Approval Number	Drug Type	Dosage Form	Approval Date
No NMPA approvals found for this drug.

PPB Drug Approvals

Approved Product	Registration No.	Company	Licence No.	Strength	Registration Date
No PPB approvals found for this drug.

TGA Drug Approvals

Approved Product	ARTG ID	Sponsor	Registration Type	Status	Registration Date
SYMDEKO 100/150 tezacaftor 100 mg/ivacaftor 150 mg film-coated tablet and ivacaftor 150 mg film-coated tablet composite pack	298329	Vertex Pharmaceuticals Australia Pty Ltd	Medicine	A	3/5/2019
TRIKAFTA 80/40/60 GRANULES elexacaftor 80 mg/tezacaftor 40 mg/ivacaftor 60 mg granules and ivacaftor 59.5 mg granules sachet composite pack	402132	Vertex Pharmaceuticals Australia Pty Ltd	Medicine	A	2/7/2024
SYMDEKO 50/75 tezacaftor 50 mg/ivacaftor 75 mg film-coated tablet and ivacaftor 75 mg film-coated tablet composite pack	337367	Vertex Pharmaceuticals Australia Pty Ltd	Medicine	A	4/29/2021
TRIKAFTA 100/50/75 GRANULES elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg granules and ivacaftor 75 mg granules sachet composite pack	402133	Vertex Pharmaceuticals Australia Pty Ltd	Medicine	A	2/7/2024
TRIKAFTA 50/25/37.5 elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg film-coated tablet and ivacaftor 75 mg film-coated tablet blister pack composite pack	372334	Vertex Pharmaceuticals Australia Pty Ltd	Medicine	A	11/1/2022
TRIKAFTA 100/50/75 elexacaftor 100 mg / tezacaftor 50 mg / ivacaftor 75 mg film-coated tablet and ivacaftor 150 mg film-coated tablet blister composite pack	330423	Vertex Pharmaceuticals Australia Pty Ltd	Medicine	A	3/24/2021

Health Canada Drug Approvals

Approved Product	Company	DIN	Dosage Form	Strength	Market Date
TRIKAFTA	Vertex Pharmaceuticals (Canada) Incorporated	02542285	Granules - Oral	40 MG	10/27/2023
TRIKAFTA	Vertex Pharmaceuticals (Canada) Incorporated	02526670	Tablet - Oral	25 MG	4/22/2022
TRIKAFTA	Vertex Pharmaceuticals (Canada) Incorporated	02517140	Tablet - Oral	50 MG	6/22/2021
SYMDEKO	Vertex Pharmaceuticals (Canada) Incorporated	02478080	Tablet - Oral	100 MG	6/28/2018
TRIKAFTA	Vertex Pharmaceuticals (Canada) Incorporated	02542277	Granules - Oral	50 MG	10/27/2023

CIMA AEMPS Drug Approvals

Approved Product	Company	Registration Number	Pharmaceutical Form	Prescription Type	Status
KAFTRIO 60 MG/40 MG/80 MG GRANULADO EN SOBRE	Vertex Pharmaceuticals (Ireland) Limited	1201468003	GRANULADO EN SOBRE	Uso Hospitalario	Commercialized
SYMKEVI 100 MG/150 MG COMPRIMIDOS RECUBIERTOS CON PELICULA	Vertex Pharmaceuticals (Ireland) Limited	1181306001	COMPRIMIDO RECUBIERTO CON PELÍCULA	Uso Hospitalario	Commercialized
KAFTRIO 37,5 MG/25 MG/50 MG COMPRIMIDOS RECUBIERTOS CON PELICULA	Vertex Pharmaceuticals (Ireland) Limited	1201468002	COMPRIMIDO RECUBIERTO CON PELÍCULA	Uso Hospitalario	Commercialized
KAFTRIO 75 MG/50 MG/100 MG COMPRIMIDOS RECUBIERTOS CON PELICULA	Vertex Pharmaceuticals (Ireland) Limited	1201468001	COMPRIMIDO RECUBIERTO CON PELÍCULA	Uso Hospitalario	Commercialized
SYMKEVI 50 mg/75 mg COMPRIMDOS RECUBIERTOS CON PELICULA	Vertex Pharmaceuticals (Ireland) Limited	1181306002	COMPRIMIDO RECUBIERTO CON PELÍCULA	Uso Hospitalario	Commercialized
KAFTRIO 75 MG/50 MG/100 MG GRANULADO EN SOBRE	Vertex Pharmaceuticals (Ireland) Limited	1201468004	GRANULADO EN SOBRE	Uso Hospitalario	Commercialized

Philippines FDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Philippines FDA approvals found for this drug.

Saudi SFDA Drug Approvals

Approved Product	Company	License Number	Dosage Form	Strength	Approval Date
No Saudi SFDA approvals found for this drug.

Malaysia NPRA Drug Approvals

Approved Product	Company	Registration Number	Dosage Form	Strength	Approval Date
No Malaysia NPRA approvals found for this drug.

UK EMC Drug Information

Medicine Name	MA Holder	MA Number	Pharmaceutical Form	Active Ingredient	Authorization Date
No UK EMC drug information found for this drug.

Related News

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New real-world evidence presented at ATS 2025 validates Trikafta (elexacaftor/tezacaftor/ivacaftor) as an effective long-term therapy for cystic fibrosis patients with at least one F508del mutation.

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TRIKAFTA Expanded by FDA to Treat Younger Cystic Fibrosis Patients and Additional Mutations