Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ELX/TEZ/IVA · 19 trials · 1 indication
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Percent reduction in cough frequency was analyzed with a mixed effects model for repeated measures (MMRM), with change from baseline at each post-baseline visit on the natural log scale as the dependent variable. The percent reduction was estimated as 100% × (1-exponential form of LS mean change estimate from the MMRM).
Baseline 2-hour post-OGTT blood glucose level was defined as the average of valid pre-dose measurements at screening and Day 1. OGTT results were considered valid only when the participant was fasting for at least 8 hours.
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
| Arm | Type | Description |
|---|---|---|
| ELX/TEZ/IVA | EXPERIMENTAL | Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening. |
| Part A | EXPERIMENTAL | Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening. |
| Part B | EXPERIMENTAL | Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening with the dose(s) to be based on the outcome of Part A. |
| Placebo | PLACEBO_COMPARATOR | Participants received placebo matched to ELX/TEZ/IVA FDC in the morning and placebo matched to IVA in the evening for 24 weeks. |
| Part A: ELX/TEZ/IVA | EXPERIMENTAL | Participants weighing greater than or equal to (\>=)14 kilograms (kg) at screening received elexacaftor (ELX) 100 milligrams (mg) once daily (qd)/tezacaftor (TEZ) 50 mg qd/ivacaftor (IVA) 75 mg every 12 hours (q12h) in the treatment period for 15 days. |
| Part B: ELX/TEZ/IVA | EXPERIMENTAL | Participants weighing (\>=)14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h. Participants weighing (\>=)10 kg to less than (\<)14 kg received ELX 80 mg qd/TEZ 40 mg qd/IVA 60 mg once every morning (qAM) and 59.5 mg once every evening (qPM) in the treatment period for 24 weeks. |
| TEZ/IVA | ACTIVE_COMPARATOR | Following TEZ/IVA run-in period of 4 weeks, participants received TEZ 100 milligrams (mg) once daily (qd)/IVA 150 mg every 12 hours (q12h) in the treatment period for 24 weeks. |
| Control: IVA or TEZ/IVA | ACTIVE_COMPARATOR | Following an IVA or TEZ/IVA run-in period of 4 weeks, participants either received IVA 150 milligrams (mg) every 12 hours (q12h) or TEZ 100 mg once daily (qd)/IVA 150 mg q12h in the treatment period for 8 weeks. |
| TC: ELX/TEZ/IVA | EXPERIMENTAL | Following an IVA or TEZ/IVA run-in period of 4 weeks, participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 8 weeks. |
| Name | Type | Description |
|---|---|---|
| ELX/TEZ/IVA | DRUG | Fixed-dose combination granules for oral administration. |
| IVA | DRUG | Granules for oral administration |
| Placebo (matched to ELX/TEZ/IVA) | OTHER | Placebo matched to ELX/TEZ/IVA for oral administration. |
| Placebo (matched to IVA) | OTHER | Placebo matched to IVA for oral administration. |
| TEZ/IVA | DRUG | Fixed-dose combination (FDC) tablet for oral administration. |
Key Inclusion Criteria: * Completed study drug treatment in the parent study VX22-445-122 Part B (NCT05882357) OR had study drug interruption(s) in the parent study but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the Treatment Period of th...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Vertex Pharmaceuticals Incorporated | VRTX | 8 | PHASE3 | VX-121/TEZ/D-IVA |
| Sionna Therapeutics, Inc. | SION | 2 | PHASE2 | SION-719 |
| 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 |
ELX/TEZ/IVA is a small molecule combination therapy used for the treatment of cystic fibrosis. It is being developed by Vertex Pharmaceuticals and is currently in Phase 3 clinical trials. The drug is being studied in patients as young as 12 months old, as well as in adults and children with various CF genotypes.
ELX/TEZ/IVA is a combination of three drugs: elexacaftor, tezacaftor, and ivacaftor. These drugs target the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is defective in people with cystic fibrosis. The combination works to improve CFTR function and reduce the symptoms of the disease.
ELX/TEZ/IVA is developed by Vertex Pharmaceuticals Incorporated, which is publicly traded under the ticker symbol VRTX. The company is conducting Phase 3 clinical trials to evaluate the safety and efficacy of this combination therapy in patients with cystic fibrosis.
ELX/TEZ/IVA is currently in Phase 3 clinical development. It is an investigational drug and has not yet been approved by regulatory authorities. Vertex Pharmaceuticals is conducting multiple Phase 3 trials to evaluate its safety and efficacy in patients with cystic fibrosis.
ELX/TEZ/IVA is being studied in several Phase 3 clinical trials, including NCT04058353, NCT05153317, NCT05331183, and NCT06460506. These trials evaluate the drug's safety and efficacy in cystic fibrosis patients of various ages and genotypes, with some trials completed and others still active.
Yes, ELX/TEZ/IVA is the abbreviation for the combination of elexacaftor, tezacaftor, and ivacaftor. This triple combination therapy is being developed by Vertex Pharmaceuticals for the treatment of cystic fibrosis and is currently in Phase 3 clinical trials.