TheraRadar
Data updated: May 26, 2026

ORKAMBI (ivacaftor)

Chloride Channel Activation Potentiators Trial Activity: Declining 1 active trials
Respiratory Approved 2015-07-02

ORKAMBI is indicated for the treatment of Cystic Fibrosis.

Source: FDA Label • Vertex Pharmaceuticals • Cystic Fibrosis Transmembrane Conductance Regulator Potentiator
Jump to: Indications Approvals Trials Competitors Similar Safety Patents

How ORKAMBI Works

The F508del mutation causes CFTR protein misfolding and degradation, leading to reduced protein quantity and defective channel gating at the cell surface. Lumacaftor improves the conformational stability of the F508del-CFTR protein, which increases the processing and trafficking of mature protein to the cell surface. Ivacaftor functions as a CFTR potentiator that increases chloride transport by improving the channel-open probability, or gating, of the protein. Together, these components increase the quantity, stability, and function of the CFTR protein to enhance chloride ion transport.

Source: FDA Label

Development Insights

Vertex Pharmaceuticals Incorporated conducting 39 trials (83%)
6 indications explored (Moderate)
→ cystic fibrosis (42 trials)
→ advanced lung disease (2 trials)
→ cystic fibrosis, homozygous for the f508del cftr mutation (2 trials)
6
Indications
--
Phase 3 Trials
5
Priority Reviews
10
Years on Market

Details

Status
Prescription
First Approved
2015-07-02
Patent Cliff
2031
Revenue
$111M (Q4-2022)

Pro Metrics

Patent cliff and revenue data

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Routes
ORAL
Dosage Forms
TABLET, GRANULE

Companies

Vertex Pharmaceuticals
Active Ingredient: IVACAFTOR , LUMACAFTOR

ORKAMBI Approval History

2016
2017
2018
2019
2020
2021
2022
2023
2024
2025
2026
Original
New Indication
New Form
Label Update
46 FDA actions from 2015 to 2026 · 3 indication expansions
Mar 2026 SUPPL
Label · Labeling
FDA Letter Label
Sep 2025 SUPPL
Label · Labeling
FDA Letter Label
Dec 2024 SUPPL Priority
Efficacy
FDA Letter Label

What ORKAMBI Treats

1 indications

ORKAMBI is approved for 1 conditions since its original approval in 2015. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Cystic Fibrosis
Source: FDA Label

ORKAMBI Competitive Set

Pro

Three rings of competition based on shared molecular targets and treated indications.

Direct competitors

1

Same target(s) AND same indication — head-to-head.

ALYFTREK
Vertex Pharmaceuticals
2024 1 indic.

MoA expansion candidates

3

Same target(s), different indications — where else is this mechanism being explored?

TRIKAFTA (COPACKAGED)
Vertex Pharmaceuticals
2019
MYTESI
NAPO PHARMS
2012 2 indic.

Indication competitors

3

Same indication, different mechanism — what else might this patient receive?

PULMOZYME
Roche
1993 2 indic.
VITAMIN K1
Pfizer
1983 10 indic.
Unlock 2 more competitors across all three rings.
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Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.

Drugs Similar to ORKAMBI

3 of 12

FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.

ALYFTREK
DEUTIVACAFTOR
1 shared
Vertex Pharmaceuticals
Shared indications:
Cystic Fibrosis
BETHKIS
TOBRAMYCIN
1 shared
CHIESI
Shared indications:
Cystic Fibrosis
CAYSTON
AZTREONAM
1 shared
Gilead Sciences
Shared indications:
Cystic Fibrosis
View all 12 similar drugs Pro
📋

Clinical Trial Registry

41 trials
Trial Sponsor ID Phase Status Title
NCT06237335 RCT2100-101 2024-512169-15 Ph 2 recruiting A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
NCT02070744 results posted VX13-661-103 Ph 2 completed Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion
NCT04126473 EL-004 Ph 2 completed A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele
NCT04135495 EL-012 Ph 2 completed A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele
NCT04066751 IRB-300003967 P30DK072482 Ph 2 withdrawn The Multicenter Topic Trial
NCT02871778 CLEAN-PCD results posted PS-G202 2015-004917-26 Ph 2 completed Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia
NCT02709109 results posted VX15-371-101 2015-004841-13 Ph 2 completed A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
NCT02508207 results posted VX14-661-111 Ph 2 completed A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
NCT03956589 2018-001573-24 Ph 4 terminated Functional Respiratory Imaging and Orkambi in CF
NCT03068312 results posted VX16-770-127 2017-000457-39 Ph 3 completed A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation
NCT02730208 results posted VX15-661-112 Ph 2 completed A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
NCT03150719 results posted VX16-661-114 2017-000540-18 Ph 3 completed A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)
NCT02412111 results posted VX14-661-109 Ph 3 completed A Phase 3 Study of Tezacaftor (VX-661) in Combination With Ivacaftor (VX-770) in Subjects Aged 12 Years and Older With Cystic Fibrosis (CF), Who Have One F508del-CFTR Mutation and a Second Mutation That Has Been Demonstrated to be Clinically Responsive to Ivacaftor
NCT02742519 results posted VX15-770-123 2015-001267-39 Ph 3 terminated A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation
NCT02934698 results posted Pro00052526 Ph 3 completed An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations
NCT02392234 results posted VX14-661-108 2014-004788-18 Ph 3 completed A Phase 3 Study to Evaluate the Efficacy and Safety of Ivacaftor and VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Mutation
NCT02347657 results posted VX14-661-106 Ph 3 completed A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor
NCT02516410 results posted VX14-661-107 2014-004787-37 Ph 3 completed A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation
NCT01531673 results posted VX11-661-101 2011-003821-93 Ph 2 completed Study of VX-661 Alone and in Combination With Ivacaftor in Subjects Homozygous or Heterozygous to the F508del-Cystic Fibrosis Transmembrane Conductance Regulator(CFTR) Mutation
NCT02390219 results posted VX14-809-106 Ph 3 completed Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease
NCT03029455 VX16-659-001 2016-003048-35 Ph 1 completed A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis
NCT01897233 results posted VX13-809-011 Ph 3 completed Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
NCT01707290 KONTINUE results posted VX12-770-112 2012-000389-39 Ph 3 completed Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation
NCT01931839 results posted VX12-809-105 Ph 3 completed A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis
NCT01946412 results posted VX11-770-109 Ph 3 completed Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation
NCT01807949 TRANSPORT results posted VX12-809-104 Ph 3 completed A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation
NCT01705145 results posted VX11-770-108 KIWI Ph 3 completed Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation
NCT01225211 results posted VX09-809-102 2010-020413-90 Ph 2 completed Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation
NCT00953706 DISCOVER results posted VX08-770-104 2009-010261-23 Ph 2 terminated Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation
NCT01807923 TRAFFIC results posted VX12-809-103 Ph 3 completed A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation
NCT01117012 results posted VX08-770-105 PERSIST Ph 3 completed Rollover Study of VX-770 in Cystic Fibrosis Subjects
NCT01685801 results posted VX12-770-113 Ph 2 completed Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function
NCT01614457 KONDUCT results posted VX11-770-110 Ph 3 completed Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H-CF Transmembrane Conductance Regulator (CFTR) Mutation (KONDUCT)
NCT01614470 KONNECTION results posted VX12-770-111 Ph 3 completed Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation
NCT01910415 VX12-809-008 Ph 1 completed Phase 1, QT/QTC Interval Study in Healthy Subjects
NCT01899105 VX13-809-012 Ph 1 completed A Phase 1 Study to Investigate the Food Effect of Lumacaftor in Combination With Ivacaftor
NCT02015507 VX13-770-017 Ph 1 completed An Open-Label, Phase 1 Study in Healthy Adult Subjects to Examine the Effects of Multiple-Dose Ciprofloxacin on Ivacaftor and VX-661 in Combination With Ivacaftor
NCT01768663 VX12-809-009 Ph 1 completed A Phase 1 Study to Examine the Drug-Drug Interaction of Ciprofloxacin, Itraconazole, and Rifampin on the Combination of Lumacaftor With Ivacaftor in Healthy Adult Subjects
NCT01262352 results posted VX10-770-106 Ph 2 completed Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation
NCT00909532 STRIVE results posted VX08-770-102 Ph 3 completed Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation
NCT00909727 ENVISION results posted VX08-770-103 Ph 3 completed Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation
🔬

Active Pipeline

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Ongoing clinical trials by development phase

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Key Completed Trials

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Completed studies with published results, ranked by significance

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Trial Timeline

ClinicalTrials.gov ↗ Pro

Full development history with FDA approval milestones

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Understanding FDA Approval Types
Count Type What it means
- ORIG Original approval - drug first enters market
- SUPPL - Efficacy New indication (new disease/condition approved)
- SUPPL - Labeling Label text changes (warnings, dosing updates)
- SUPPL - Manufacturing Production changes (new facility)
- SUPPL - Chemistry Formulation changes (new dosage strength)

Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.

ORKAMBI FDA Label Details

Indications & Usage

FDA Label (PDF)

ORKAMBI is indicated for the treatment of Cystic Fibrosis.

View full patent landscape →
46 OB patents · 8 families · 539 international docs across 37 countries

ORKAMBI Patents & Exclusivity

Latest Patent: Jun 2031
Exclusivity: Mar 2030

Patents (46 active)

US8993600*PED Expires Jun 11, 2031
US8507534*PED Expires Mar 20, 2031
US8716338*PED Expires Mar 20, 2031
US8993600 Expires Dec 11, 2030
US8716338 Expires Sep 20, 2030
US8507534 Expires Sep 20, 2030
US9192606*PED Expires Mar 29, 2030
US10646481*PED Expires Feb 13, 2030
US12458635*PED Expires Feb 13, 2030
US11564916*PED Expires Feb 13, 2030
US8846718*PED Expires Jan 2, 2030
US9192606 Expires Sep 29, 2029
US12458635 Expires Aug 13, 2029
US11564916 Expires Aug 13, 2029
US10646481 Expires Aug 13, 2029
US8846718 Expires Jul 2, 2029
US8653103*PED Expires Jun 4, 2029
US9150552*PED Expires Jun 4, 2029
US10076513*PED Expires Jun 4, 2029
US11052075*PED Expires Jun 4, 2029
US12065432*PED Expires Jun 4, 2029
US10597384*PED Expires Jun 4, 2029
US9150552 Expires Dec 4, 2028
US10076513 Expires Dec 4, 2028
US10597384 Expires Dec 4, 2028
US12065432 Expires Dec 4, 2028
US11052075 Expires Dec 4, 2028
US8653103 Expires Dec 4, 2028
US8324242*PED Expires Feb 5, 2028
US7495103*PED Expires Nov 20, 2027
US8324242 Expires Aug 5, 2027
US8410274*PED Expires Jun 28, 2027
US8754224*PED Expires Jun 28, 2027
US9670163*PED Expires Jun 28, 2027
US9931334*PED Expires Jun 28, 2027
US7495103 Expires May 20, 2027
US9216969*PED Expires May 8, 2027
US8741933*PED Expires May 8, 2027
US7973038*PED Expires May 8, 2027
US9931334 Expires Dec 28, 2026
US9670163 Expires Dec 28, 2026
US8754224 Expires Dec 28, 2026
US8410274 Expires Dec 28, 2026
US7973038 Expires Nov 8, 2026
US8741933 Expires Nov 8, 2026
US9216969 Expires Nov 8, 2026

Exclusivity

M-14 Until Dec 2027
ODE-408 Until Sep 2029
M-14 Until Dec 2027
ODE-408 Until Sep 2029
M-14 Until Dec 2027
ODE-408 Until Sep 2029
PED Until Mar 2030
PED Until Mar 2030
PED Until Mar 2030
PED Until Jun 2028
PED Until Jun 2028
PED Until Jun 2028
M-14 Until Dec 2027
ODE-408 Until Sep 2029
M-14 Until Dec 2027
ODE-408 Until Sep 2029
M-14 Until Dec 2027
ODE-408 Until Sep 2029
PED Until Mar 2030
PED Until Mar 2030
PED Until Mar 2030
PED Until Jun 2028
PED Until Jun 2028
PED Until Jun 2028
M-14 Until Dec 2027
ODE-408 Until Sep 2029
M-14 Until Dec 2027
ODE-408 Until Sep 2029
M-14 Until Dec 2027
ODE-408 Until Sep 2029
PED Until Mar 2030
PED Until Mar 2030
PED Until Mar 2030
PED Until Jun 2028
PED Until Jun 2028
PED Until Jun 2028
Source: FDA Orange Book

Pro Intelligence Preview

Deep insights for ORKAMBI

Revenue Insights

  • • Q4-2022: $111M
  • • Historical trend analysis

Patent Timeline

  • • Cliff: 2031
  • • 566 active patents

Trial Analysis

  • • 47 total trials
  • • Stage: Declining

Competitive Landscape

  • • 12 similar drugs
  • • Same target/indication analysis
Unlock Full Intelligence

Full approval history • All patents • Revenue trends • Competitor analysis

Data Sources

FDA Approval: NDA206038 → Label: DailyMed → Patents: FDA Orange Book → Trials: ClinicalTrials.gov →

Data sourced from official FDA and NIH databases. Click links to verify on original sources.

How We Calculate These Metrics

Trial Activity Stage

Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.

Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.

  • Growth: High proportion of early-phase trials (Phase 1/2), active development
  • Expansion: Significant Phase 3 activity, approaching or pursuing approvals
  • Mature: Substantial Phase 4 post-marketing studies
  • Stable: Mixed phase distribution, steady development
  • Declining: Low active trial ratio, reduced R&D investment