Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Also known as TEZ
Tezacaftor · 8 trials · 1 indication
LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
RAESIs included chest discomfort, dyspnea (shortness of breath), respiration abnormal (chest tightness), asthma, bronchial hyperreactivity, bronchospasm, and wheezing.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
The exploratory Brody/CF-CT score semi-quantitatively scores the degree of structural lung disease as shown on CT in participants with CF. The score ranges from a minimum of 0 to a maximum of 219 with higher scores indicating more severe structural lung disease.
MCC was assessed using an imaging technique that enables the tracking of mucus within the airways. MCC was expressed as the percentage of whole-lung clearance through 60 minutes at Baseline and Day 28.
| Arm | Type | Description |
|---|---|---|
| Placebo | OTHER | Participants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks. |
| TEZ/IVA | EXPERIMENTAL | Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks. |
| Ivacaftor | EXPERIMENTAL | Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks. |
| Part A | EXPERIMENTAL | Participants weighing \<25 kg received TEZ 50 mg once daily/IVA 75 mg q12h orally for 14 days. Participants weighing ≥25 kg received TEZ 50 mg once daily/IVA 150 mg q12h orally for 14 days. |
| Part B | EXPERIMENTAL | Participants weighing \<40 kg received TEZ 50 mg/IVA 75 mg as fixed dose combination orally once daily in the morning and IVA 75 mg orally once daily in the evening for 24 weeks. Participants weighing ≥40 kg received TEZ 100 mg/IVA 150 mg as fixed dose combination orally once daily in the morning and IVA 150 mg orally once daily in the evening for 24 weeks. |
| Part 1: Placebo - Cohort 1A and 1B Combined | PLACEBO_COMPARATOR | Participants received placebo matched to VX-440/TEZ/IVA as triple combination for 4 weeks. |
| Part 1 Cohort 1A: Triple Combination (TC) | EXPERIMENTAL | Participants received VX-440 200 milligram (mg) every 12 hours (q12h)/TEZ 100 mg once daily (qd)/IVA 150 mg q12h as triple combination for 4 weeks. |
| Part 1 Cohort 1B: TC Low Dose | EXPERIMENTAL | Participants received VX-440 200 mg q12h/TEZ 50 mg q12h/IVA 150 mg q12h as triple combination for 4 weeks. |
| Part 1 Cohort 1B: TC High Dose | EXPERIMENTAL | Participants received VX-440 600 mg q12h/TEZ 50 mg q12h/IVA 300 mg q12h as triple combination for 4 weeks. |
| Part 2: TEZ/IVA | ACTIVE_COMPARATOR | Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received placebo matched to VX-440 and TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period. |
| Part 2: TC-2 | EXPERIMENTAL | Following a 4-week run-in period on TEZ 100 mg qd/IVA150 mg q12h, participants received VX-440 600 mg q12h/ TEZ 50 mg q12h/IVA 300 mg q12h for 4 weeks for 4 weeks in treatment period and TEZ 100 mg qd/IVA150 mg q12h for 4 weeks in washout period. |
| Name | Type | Description |
|---|---|---|
| TEZ/IVA | DRUG | Participants weighing \<40 kg received TEZ 50 mg/IVA 75 mg FDC tablet and those weighing ≥40 kg received TEZ 100 mg/IVA 150 mg FDC tablet. |
| IVA | DRUG | Participants weighing \<40 kg IVA 75 mg tablet and those weighing ≥40 kg received IVA 150 mg tablet. |
| Placebo | DRUG | Placebo matched to TEZ/IVA FDC |
| Tezacaftor/Ivacaftor | DRUG | TEZ 100 mg/IVA 150 mg fixed-dose combination tablet. |
| Ivacaftor | DRUG | IVA 150 mg tablet. |
| TEZ | DRUG | - |
| VX-440 | DRUG | - |
| Matched Placebo | DRUG | - |
| Tezacaftor/Ivacaftor matching placebo | DRUG | - |
| Ivacaftor matching placebo | DRUG | - |
Key Inclusion Criteria: * Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol). * Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height. * Participants with a screening LCI2.5 result ≥7.5. * Participants who are able to swallow...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | VX-121/TEZ/D-IVA |
| BiomX Inc. | PHGE | 1 | PHASE2 | BX004 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-710 |
| Arcturus Therapeutics Holdings, Inc. | ARCT | 1 | PHASE2 | ARCT-032 |
| Krystal Biotech, Inc. | KRYS | 1 | PHASE1 | KB407 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
Tezacaftor/Ivacaftor is used for the treatment of cystic fibrosis. It is a small molecule combination therapy being developed by Vertex Pharmaceuticals Incorporated. The drug is intended for patients with cystic fibrosis, including those who have previously been treated with Orkambi (lumacaftor/ivacaftor).
Tezacaftor/Ivacaftor is a combination of two small molecules that target the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Tezacaftor is a CFTR corrector that helps the protein fold properly, while ivacaftor is a potentiator that enhances channel function. Together, they improve chloride transport in patients with the F508del-CFTR mutation.
Tezacaftor/Ivacaftor is developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. Vertex is the sole developer of this combination therapy for cystic fibrosis.
Tezacaftor/Ivacaftor is in Phase 3 clinical development. It has completed three clinical trials, including one Phase 3 study. The drug is investigational and has not been approved by the FDA, as it is still undergoing clinical evaluation for cystic fibrosis.
Tezacaftor/Ivacaftor has completed three clinical trials. NCT02508207 was a Phase 2 study in the United States with 34 participants. NCT02730208 was a Phase 2 study in Australia with 41 participants. NCT03150719 was a Phase 3 study in the United States, France, and Germany with 98 participants.
Tezacaftor/Ivacaftor is not the same as Orkambi. Orkambi is a combination of lumacaftor and ivacaftor, while Tezacaftor/Ivacaftor combines tezacaftor with ivacaftor. The Phase 3 trial NCT03150719 specifically evaluated Tezacaftor/Ivacaftor in patients who had previously been treated with Orkambi.