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ELX/TEZ/IVA

Phase 3

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: Jun 5, 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
RandomizedDouble-BlindACTIVE_CONTROLLEDDMCBiomarker
Total Trials19
Total Enrollment3,326
FDA Designations
No designations recorded
Clinical Trials (19)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06460506Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and OlderPHASE3 ACTIVE NOT_RECRUITING 50 — — Nov 21, 2024Sep 30, 2027Jun 5, 202618 Australia, Canada +5
NCT05882357Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of AgePHASE3 COMPLETED 70 — — Jun 27, 2023Sep 4, 2025Oct 6, 202519 Australia, Canada +5
NCT05331183Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508delPHASE3 ACTIVE NOT_RECRUITING 297 — — Nov 23, 2022Apr 6, 2027May 29, 202681 Austria, Belgium +13
NCT05274269Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del MutationPHASE3 COMPLETED 307 — — May 9, 2022Jul 5, 2023Aug 1, 202490 Austria, Belgium +13
NCT05153317Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and OlderPHASE3 COMPLETED 71 — — Jan 17, 2022Jan 29, 2026Mar 3, 202622 United States, Australia +3
NCT05111145A Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor in Participants With Cystic Fibrosis (CF)PHASE3 COMPLETED 86 — — Jan 14, 2022Dec 20, 2022Jul 11, 202324 Australia, Belgium +3
NCT04969224A Study to Evaluate ELX/TEZ/IVA on Cough and Physical Activity in Participants With Cystic Fibrosis (CF)PHASE3 COMPLETED 82 — — Oct 12, 2021Jul 26, 2022Sep 24, 202519 Australia, Belgium +2
NCT04599465A Study to Assess the Effect of ELX/TEZ/IVA on Glucose Tolerance in Participants With Cystic Fibrosis (CF)PHASE3 COMPLETED 69 — — Jan 15, 2021Jul 14, 2022Aug 3, 202341 Australia, Belgium +5
NCT04545515A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF GenotypesPHASE3 COMPLETED 120 — — Jan 11, 2021Mar 24, 2023May 8, 202434 Australia, Canada +8
NCT04537793Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 YearsPHASE3 COMPLETED 83 — — Nov 19, 2020Jun 3, 2022Jun 28, 202322 United States, Australia +3
NCT04353817A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF GenotypesPHASE3 COMPLETED 121 — — Jun 19, 2020May 17, 2021Jul 26, 202234 Australia, Canada +8
NCT04362761A Study Evaluating the Long-Term Safety of Elexacaftor Combination TherapyPHASE3 COMPLETED 172 — — May 4, 2020Dec 21, 2022Jul 28, 202329 Australia, Belgium +2
NCT04183790Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and OlderPHASE3 COMPLETED 64 — — Feb 17, 2020Feb 24, 2024May 18, 202521 United States, Australia +3
NCT04058366Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyPHASE3 COMPLETED 251 — — Dec 5, 2019Dec 16, 2022Jan 16, 202484 United States, Australia +10
NCT04105972A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508delPHASE3 COMPLETED 176 — — Oct 3, 2019Jul 24, 2020Aug 18, 202135 Australia, Belgium +2
NCT04058353A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)PHASE3 COMPLETED 271 — — Aug 28, 2019Jun 12, 2020Jul 2, 202193 United States, Australia +11
NCT04043806A Study Evaluating the Long-term Safety of VX-445 Combination TherapyPHASE3 COMPLETED 458 — — Aug 9, 2019Dec 14, 2022Jul 6, 202397 United States, Australia +9
NCT03525574A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination TherapyPHASE3 COMPLETED 507 — — Oct 9, 2018Jan 9, 2023Sep 24, 2025110 United States, Australia +11
NCT03691779Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of AgePHASE3 COMPLETED 71 — — Oct 2, 2018Aug 7, 2020Oct 22, 202121 United States, Australia +3
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Study Endpoints
Primary Endpoints
Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 100
Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites
Day 1 up to Day 15
Part A: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to Day 43
Part B: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 28
Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 196
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
From Baseline Through Week 24

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 36
Percent Reduction From Baseline in Cough Frequency (Cough Events Per Day) to the Average of Week 8 Through Week 12
Baseline, Week 8 through Week 12

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).

Change From Baseline in 2-hour Blood Glucose Levels Following an OGTT to the Average of Week 36 and Week 48
Baseline, Week 36 and 48

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.

Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
From Day 1 through Day 15
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Day 1 up to Day 43
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Day 1 up to Week 28
Absolute Change in Lung Clearance Index 2.5 (LCI2.5)
From Baseline Through Week 24

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.

Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Day 1 up to Week 86
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Baseline up to Week 196
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Baseline up to Week 100
Absolute Change in CF Questionnaire-Revised (CFQ-R) Respiratory Domain Score
From Baseline Through Week 24

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.

Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) for ELX/TEZ/IVA Group
From Baseline Through Week 8

FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.

Treatment Period: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
From Day 1 up to Week 196
Part A: Maximum Observed Plasma Concentration (Cmax) of ELX, TEZ, and IVA
Part A: Day 15
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of ELX, TEZ, and IVA
Part A: Day 15
Part A: Area Under the Concentration Versus Time Curve From 0 to 24 Hours (AUC0-24h) of ELX, TEZ, and IVA
Part A: Day 15
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Part B: Day 1 Through Safety Follow-up Visit (up to Week 28)
Secondary Endpoints
Absolute Change in Sweat Chloride (SwCl)
From Baseline through Week 96
Part B: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites
Day 15 up to Week 16
Part B: Absolute Change in Sweat Chloride (SwCl)
From Baseline Through Week 24
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ELX/TEZ/IVAEXPERIMENTALParticipants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Part AEXPERIMENTALParticipants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Part BEXPERIMENTALParticipants 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.
PlaceboPLACEBO_COMPARATORParticipants 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/IVAEXPERIMENTALParticipants 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/IVAEXPERIMENTALParticipants 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/IVAACTIVE_COMPARATORFollowing 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/IVAACTIVE_COMPARATORFollowing 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/IVAEXPERIMENTALFollowing 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.
Interventions
NameTypeDescription
ELX/TEZ/IVADRUGFixed-dose combination granules for oral administration.
IVADRUGGranules for oral administration
Placebo (matched to ELX/TEZ/IVA)OTHERPlacebo matched to ELX/TEZ/IVA for oral administration.
Placebo (matched to IVA)OTHERPlacebo matched to IVA for oral administration.
TEZ/IVADRUGFixed-dose combination (FDC) tablet for oral administration.
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Eligibility Criteria
Age Range12 Months — N/A
SexALL
Healthy VolunteersNo
Study Sites18

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...

Countries:AustraliaCanadaDenmarkGermanyNetherlandsSwitzerlandUnited KingdomAustriaBelgiumCzechiaFranceHungaryItalyNorwayPolandPortugalSpainSwedenUnited StatesIsraelIrelandGreece
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Competitive Landscape -Cystic Fibrosis 18 trials
CompanyTickerTrialsLead PhaseDrugs
Vertex Pharmaceuticals IncorporatedVRTX9PHASE3VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, VNZ/TEZ/D-IVA, VX-522 mRNA therapy
Sionna Therapeutics, Inc.SION2PHASE2SION-719, SION-451, SION-2222, SION-109
BiomX LtdPHGE1PHASE2BX004
4D Molecular Therapeutics, Inc.FDMT1PHASE24D-710
Arcturus Therapeutics Holdings, Inc.ARCT1PHASE2ARCT-032
Krystal Biotech, Inc.KRYS1PHASE1KB407
Illumina, Inc.ILMN1 — Undisclosed
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Recent Changes (Last 90 Days)
LOWJun 5, 2026NCT06460506lastUpdatePostDate: changed
LOWJun 5, 2026NCT06460506lastUpdatePostDate: changed
LOWJun 5, 2026NCT06460506lastUpdatePostDate: changed
LOWJun 5, 2026NCT06460506lastUpdatePostDate: changed
LOWMay 29, 2026NCT05331183lastUpdatePostDate: changed
LOWMay 29, 2026NCT05331183lastUpdatePostDate: changed
LOWMay 29, 2026NCT05331183lastUpdatePostDate: changed
LOWMay 26, 2026NCT06460506primaryCompletionDate: changed
LOWMay 26, 2026NCT05331183primaryCompletionDate: changed
LOWMay 24, 2026NCT06460506studyFirstPostDate: changed
LOWMay 24, 2026NCT05331183studyFirstPostDate: changed
MEDIUMApr 8, 2026NCT05153317TRIAL_REMOVED: changed
MEDIUMApr 8, 2026NCT05153317TRIAL_REMOVED: changed