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VNZ/TEZ/D-IVA

Phase 3

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: Apr 29, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedCONTROLLEDBiomarker
Total Trials4
Total Enrollment236
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05844449Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and OlderPHASE3 ENROLLING BY_INVITATION 174Aug 11, 2023Jul 30, 2029Apr 29, 202637 United States, Australia +8
NCT07349394Effect of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) on the PK of Rosuvastatin in Healthy ParticipantsPHASE1 COMPLETED 18Jan 12, 2026Mar 12, 2026Apr 2, 20261 United States
NCT06299696A Study of Vanzacaftor/Tezacaftor/Deutivacaftor (VNZ/TEZ/D-IVA) in Healthy Adult PanelistsPHASE1 COMPLETED 10Mar 26, 2024Apr 16, 2024May 16, 20241 United States
NCT06299709A Study To Evaluate the Relative Bioavailability, Food Effect, and Dose Proportionality of a Granule Formulation of Vanzacaftor/Tezacaftor/Deutivacaftor(VNZ/TEZ/D-IVA)PHASE1 COMPLETED 34Mar 13, 2024May 23, 2024Sep 26, 20251 United States
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Study Endpoints
Primary Endpoints
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
From Baseline up to Week 100
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
From Baseline up to Week 196
Maximum Observed Concentration (Cmax) of Rosuvastatin in the Absence and Presence of VNZ/TEZ/D-IVA
Day 1 up to Day 23
Area Under the Concentration Time-curve (AUC) of Rosuvastatin in the Absence and Presence of VNZ/TEZ/D-IVA
Day 1 up to Day 23
Flavor Assessment Based on Flavor Profile Method of Descriptive Sensory Analysis per American Society for Testing and Material (ASTM) Approved Sensory Evaluation Method
Periodic Intervals up to 30 minutes post oral administration
Part A: Maximum Observed Plasma Concentration (Cmax) of VNZ, TEZ, and D-IVA
Pre-dose up to 288 hours Post-dose
Part B: Maximum Observed Plasma Concentration (Cmax) of VNZ, TEZ, and D-IVA
Pre-dose up to 384 hours Post-dose
Part A: Area Under the Concentration Versus Time Curve (AUC) of VNZ, TEZ, and D-IVA
Pre-dose up to 288 hours Post-dose
Part B: Area Under the Concentration Versus Time Curve (AUC) of VNZ, TEZ, and D-IVA
Pre-dose up to 384 hours Post-dose
Secondary Endpoints
Part A (All Cohorts): Absolute Change in Sweat Chloride (SwCl)
From Baseline Through Week 96
Part B: Absolute Change in Sweat Chloride (SwCl)
From Baseline Through Week 192
Part A (Cohort 1): Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)
From Baseline Through Week 100
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
VNZ/TEZ/D-IVAEXPERIMENTALPart A: Participants (Cohort 1: 6 through 11 years of age (inclusive); Cohort 2: 2 through 5 years of age (inclusive) and Cohort 3: 1 to less than (\<) 2 years of age) will receive VNZ/TEZ/D-IVA once daily at doses determined based on their age and weight. The age range is based on date of informed consent in parent study. Part B: Participants in Cohort 1 who meet the eligibility criteria will receive VNZ/TEZ/D-IVA for an additional 96 weeks.
VNZ/TEZ/D-IVA and RosuvastatinEXPERIMENTALRosuvastatin will be administered to participants as a single oral dose on Day 1 and then co-administered with VNZ/TEZ/D-IVA on Day 18.VNZ/TEZ/D-IVA dose will be administered once daily (qd) from Day 5 to Day 22.
Part A: Sequence 1EXPERIMENTALParticipants will receive VNZ/TEZ/D-IVA reference fixed dose combination (FDC) tablet in dosing period 1, then VNZ/TEZ/D-IVA test FDC granules dose level 1 in dosing period 2, and finally VNZ/TEZ/D-IVA test FDC granules dose level 2 in dosing period 3. A washout period of 14 days will be maintained between 3 dosing periods.
Part A: Sequence 2EXPERIMENTALParticipants will receive VNZ/TEZ/D-IVA test FDC granules dose level 1 in dosing period 1, then VNZ/TEZ/D-IVA test FDC granules dose level 2 in dosing period 2, and finally VNZ/TEZ/D-IVA reference FDC tablet in dosing period 3. A washout period of 14 days will be maintained between 3 dosing periods.
Part A: Sequence 3EXPERIMENTALParticipants will receive VNZ/TEZ/D-IVA test FDC granules dose level 2 in dosing period 1, then VNZ/TEZ/D-IVA reference FDC tablet in dosing period 2, and finally VNZ/TEZ/D-IVA test FDC granules dose level 1 in dosing period 3. A washout period of 14 days will be maintained between 3 dosing periods.
Part B: Sequence 1EXPERIMENTALParticipants will receive VNZ/TEZ/D-IVA test FDC granules under fasted condition in dosing period 1, then VNZ/TEZ/D-IVA test FDC granules under fed state in dosing period 2. A washout period of 18 days will be maintained between 2 dosing periods.
Part B: Sequence 2EXPERIMENTALParticipants will receive VNZ/TEZ/D-IVA test FDC granules under fed state in dosing period 1, then VNZ/TEZ/D-IVA test FDC granules under fasted condition in dosing period 2. A washout period of 18 days will be maintained between 2 dosing periods.
Interventions
NameTypeDescription
VNZ/TEZ/D-IVADRUGFixed-dose combination tablets or granules for oral administration.
RosuvastatinDRUGTablet for oral administration.
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Eligibility Criteria
Age Range1 Year — N/A
SexALL
Healthy VolunteersNo
Study Sites37

Key Inclusion Criteria: Parts A and B: • Participants who have completed study drug treatment in the parent study (VX21-121-105; NCT Number: NCT05422222) Part B: -Meets at least 1 of the following criteria: * Completed study drug treatment in Part A * Had study drug interruption(s) in Part A, b...

Countries:United StatesAustraliaCanadaFranceGermanyNetherlandsNew ZealandSwedenSwitzerlandUnited Kingdom
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Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT07349394TRIAL_REMOVED: changed
LOWMay 26, 2026NCT05844449primaryCompletionDate: changed
LOWMay 24, 2026NCT05844449studyFirstPostDate: changed
LOWMay 24, 2026NCT07349394studyFirstPostDate: changed