Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Tezacaftor/Ivacaftor · 3 trials · 1 indication
RAESIs included chest discomfort, dyspnea (shortness of breath), respiration abnormal (chest tightness), asthma, bronchial hyperreactivity, bronchospasm, and wheezing.
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 | PLACEBO_COMPARATOR | Participants received placebo matched to TEZ/IVA fixed-dose combination tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 56 days. |
| TEZ/IVA | EXPERIMENTAL | Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed-dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 56 days. |
| Name | Type | Description |
|---|---|---|
| Tezacaftor/Ivacaftor | DRUG | TEZ 100 mg/IVA 150 mg fixed-dose combination tablet. |
| Ivacaftor | DRUG | IVA 150 mg tablet. |
| Placebo | DRUG | Placebo matched to TEZ/IVA fixed-dose combination tablet. |
| Tezacaftor/Ivacaftor matching placebo | DRUG | - |
| Ivacaftor matching placebo | DRUG | - |
Inclusion Criteria: * Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures. * Prior discontinuation of Orkambi, with at least 1 respiratory sign or symptom considered related to therapy. * Resolu...
| 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 |
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.