Overview
Elexacaftor (previously VX-445) is a small molecule, next-generation corrector of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. It received FDA approval in October 2019 in combination with tezacaftor and ivacaftor as the combination product Trikafta. Elexacaftor is considered a next-generation CFTR corrector as it possesses both a different structure and mechanism as compared to first generation correctors like tezacaftor. While dual corrector/potentiator combination therapy has proven useful in the treatment of a subset of CF patients, their use is typically limited to patients who are homozygous for the F508del-CFTR gene. Elexacaftor, along with VX-659, was designed to fill the need for an efficacious CF therapy for patients who are heterozygous for F508del-CFTR and a gene that does not produce protein or produces proteins unresponsive to ivacaftor or tezacaftor. The triple combination product Trikafta, manufactured by Vertex Pharmaceuticals, is the first product approved for the treatment of CF in individuals who are either homo- or heterozygous for the F508del-CFTR gene - this represents approximately 70-90% of all CF patients.
Indication
Elexacaftor, in combination with ivacaftor and tezacaftor as the combination product Trikafta, is indicated for the treatment of cystic fibrosis (CF) in patients 12 years of age and older who have at least one F508del mutation in the CTFR gene.
Associated Conditions
- Cystic Fibrosis (CF)
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/08/29 | Not Applicable | Recruiting | |||
2024/06/14 | Phase 3 | ENROLLING_BY_INVITATION | |||
2023/05/31 | Phase 3 | Active, not recruiting | |||
2023/01/26 | N/A | Active, not recruiting | |||
2022/10/31 | N/A | Active, not recruiting | CHC Montlegia | ||
2022/10/12 | N/A | Recruiting | University of Erlangen-Nürnberg Medical School | ||
2022/04/15 | Phase 3 | Active, not recruiting | |||
2022/03/10 | Phase 3 | Completed | |||
2021/12/10 | Phase 3 | Active, not recruiting | |||
2021/11/08 | Phase 3 | Completed |
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 |
|---|---|---|---|
Authorised | 8/21/2020 | ||
Authorised | 8/21/2020 |
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 |
|---|---|---|---|---|---|
| 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 | Medicine | A | 2/7/2024 | |
| TRIKAFTA 100/50/75 GRANULES elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg granules and ivacaftor 75 mg granules sachet composite pack | 402133 | 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 | 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 | 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 | 80 MG | 10/27/2023 |
| TRIKAFTA | Vertex Pharmaceuticals (Canada) Incorporated | 02526670 | Tablet - Oral | 50 MG | 4/22/2022 |
| TRIKAFTA | Vertex Pharmaceuticals (Canada) Incorporated | 02517140 | Tablet - Oral | 100 MG | 6/22/2021 |
| TRIKAFTA | Vertex Pharmaceuticals (Canada) Incorporated | 02542277 | Granules - Oral | 100 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 | 1201468003 | GRANULADO EN SOBRE | Uso Hospitalario | Commercialized | |
| KAFTRIO 37,5 MG/25 MG/50 MG COMPRIMIDOS RECUBIERTOS CON PELICULA | 1201468002 | COMPRIMIDO RECUBIERTO CON PELÍCULA | Uso Hospitalario | Commercialized | |
| KAFTRIO 75 MG/50 MG/100 MG COMPRIMIDOS RECUBIERTOS CON PELICULA | 1201468001 | COMPRIMIDO RECUBIERTO CON PELÍCULA | Uso Hospitalario | Commercialized | |
| KAFTRIO 75 MG/50 MG/100 MG GRANULADO EN SOBRE | 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. | |||||
Help Us Improve
Your feedback helps us provide better drug information and insights.