Kalydeco

(ivacaftor)

ApprovedSmall molecule

Ivacaftor is used to treat cystic fibrosis, a genetic disease that damages the lungs and digestive system. It works by helping a defective protein in the body function better, which improves breathing and lung function in certain patients with specific genetic mutations.[2] Kalydeco is currently approved and in use across 11 indications[4].[1]

Kalydeco is one of 211 small molecule programs targeting Cystic Fibrosis. It is currently in Approved.

Indications

Total Trials

Active Trials

210

Competitors

FDA Applications

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[4][1]

Indication

Cystic FibrosisCompleted

Respiratory System DiseaseActive, not recruiting

Acute Lung InjuryActive, not recruiting

Chronic PancreatitisActive, not recruiting

SinusitisApproved

Chronic Obstructive Pulmonary DiseaseApproved

Prolonged QT IntervalApproved

Liver DiseaseApproved

Familial Long QT SyndromeApproved

Chronic BronchitisWithdrawn

Cystic FibrosisApproved

Completed

Respiratory System DiseaseApproved

Active, not recruiting

Acute Lung InjuryApproved

Active, not recruiting

Chronic PancreatitisPreclinical

Active, not recruiting

SinusitisApproved

Approved

Chronic Obstructive Pulmonary DiseaseApproved

Approved

Prolonged QT IntervalApproved

Approved

Liver DiseaseApproved

Approved

Familial Long QT SyndromeApproved

Approved

Chronic BronchitisApproved

Withdrawn

Showing 10 of 11 indications

Competition

Competitive Landscape

120 competitors in Chronic Obstructive Pulmonary Disease

#DrugCompanyPhaseTrials

Kalydeco

Vertex Pharmaceuticals

Approved

57 2

EXDENSUR

GGlaxo Dba Glaxosmithkline

Phase 3

2 3

Lunsekimig

Sanofi

Phase 3

3 4

Tozorakimab

AstraZeneca

Phase 3

2 5

CM310

KKeymed Biosciences Co.Ltd

Phase 3

1 6

Sildenafil ODT

PPfizer's Upjohn has merged with Mylan to form Viatris Inc.

Phase 2

2 7

Astegolimab

Roche

Phase 3

3 8

Titrated Oxygen

RRigshospitalet, Denmark

Phase 3

1 9

HSK39004

Haisco Pharmaceutical Group Co., Ltd.

Phase 2

2 10

Calciferol

Jazz Pharmaceuticals

Preclinical

—

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 2Active, not recruiting

Cystic Fibrosis

NCT06237335

Q4 2027

Recruiting

Cystic Fibrosis

NCT04509050

Q2 2028

ApprovedNot yet recruiting

Cystic Fibrosis (CF)

NCT07809867

Q2 2029

Recruiting

Cystic Fibrosis

NCT06191640

Clinical

Trials by Indication[1]

Cystic Fibrosis38 trials

A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT03068312·38 enrolled

Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT01897233·62 enrolled

Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have the R117H-CF Transmembrane Conductance Regulator (CFTR) Mutation (KONDUCT)

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT01614457·70 enrolled

Show 35 more trials

A Study to Evaluate Safety and Pharmacokinetics of VX-659 in Healthy Subjects and in Adults With Cystic Fibrosis

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 1·NCT03029455·163 enrolled

Observational Study of Outcomes in Cystic Fibrosis Patients With Selected Gating Mutations on a CFTR Allele (The VOCAL Study)

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals··NCT02445053·75 enrolled

Rollover Study of VX-770 in Cystic Fibrosis Subjects

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT01117012·192 enrolled

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

Terminated· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT02742519·14 enrolled

Study of VX-809 Alone and in Combination With VX-770 in Cystic Fibrosis (CF) Patients Homozygous or Heterozygous for the F508del-CFTR Mutation

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 2·NCT01225211·312 enrolled

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT00909727·52 enrolled

A Phase 3 Rollover Study of Lumacaftor in Combination With Ivacaftor in Subjects 12 Years and Older With Cystic Fibrosis

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT01931839·1,164 enrolled

Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 2·NCT01685801·24 enrolled

Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients

Completed·UUniversity of California, San Francisco·N/A·NCT01784419·10 enrolled

Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT01707290·125 enrolled

Study of the Effect of Ivacaftor on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 2·NCT01262352·21 enrolled

A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele

Completed·EEloxx Pharmaceuticals, Inc.·Phase 2·NCT04126473·17 enrolled

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

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT02392234·248 enrolled

Safety Study of Ivacaftor in Subjects With Cystic Fibrosis

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 2·NCT00457821·39 enrolled

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT00909532·167 enrolled

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

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 2·NCT02508207·34 enrolled

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

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT01807949·563 enrolled

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

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 2·NCT01531673·194 enrolled

Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT01614470·39 enrolled

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

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT02516410·168 enrolled

Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT01705145·35 enrolled

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Homozygous for the F508del-CFTR Mutation

Terminated· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 2·NCT00953706·140 enrolled

A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele

Completed·EEloxx Pharmaceuticals, Inc.·Phase 2·NCT04135495·17 enrolled

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

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 2·NCT02070744·40 enrolled

An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations

Completed·MMedical University of South Carolina·Phase 3·NCT02934698·2 enrolled

A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT03150719·98 enrolled

Real-life Follow-up of Cystic Fibrosis Patients Treated With Ivacaftor+Lumacaftor (Orkambi*)

Completed·AAssistance Publique - Hôpitaux de Paris··NCT03475381·852 enrolled

Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT01946412·33 enrolled

An Ocular Safety Study of Ivacaftor-Treated Pediatric Patients 11 Years of Age or Younger With Cystic Fibrosis

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals··NCT01863238·95 enrolled

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

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT02412111·156 enrolled

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

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT01807923·559 enrolled

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

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 2·NCT02730208·41 enrolled

Sinus Disease in Young Children With Cystic Fibrosis

Recruiting·UUniversity of California, Los Angeles··NCT06191640·80 enrolled

A Phase 1/2, Multicenter Study Evaluating the Safety, Tolerability, and Biodistribution of RCT2100 With Single-Ascending Doses in Healthy Participants and Multiple-Ascending Doses and Proof-of-Concept in Participants With Cystic Fibrosis

Active, not recruiting·RReCode Therapeutics·Phase 2·NCT06237335·128 enrolled

A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)

Recruiting·SSonya Heltshe··NCT04509050·210 enrolled

Adult Subject2 trials

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

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 1·NCT02015507·34 enrolled

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

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 1·NCT01768663·80 enrolled

Phase 12 trials

A Phase 1 Study to Investigate the Food Effect of Lumacaftor in Combination With Ivacaftor

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 1·NCT01899105·28 enrolled

Phase 1, QT/QTC Interval Study in Healthy Subjects

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 1·NCT01910415·200 enrolled

Treatment2 trials

Ivacaftor Treatment in 4 Month to 2 Year Old CF Subjects

Completed·—··NCT03783286·15 enrolled

Novel Therapeutic Approaches for Treatment of CF Patients With W1282X Premature Termination Codon Mutations

Terminated·UUniversity of Alabama at Birmingham·Phase 4·NCT03624101·1 enrolled

Trial2 trials

The Multicenter Topic Trial

Withdrawn·UUniversity of Alabama at Birmingham·Phase 2·NCT04066751

TOPIC Trial for COPD

Completed·UUniversity of Alabama at Birmingham·Phase 1·NCT02135432·12 enrolled

11 more indications

Chronic Rhinosinusitis1 trial

Ivacaftor for Acquired CFTR Dysfunction in Chronic Rhinosinusitis

Recruiting·UUniversity of Alabama at Birmingham·Phase 1·NCT03439865·20 enrolled

Chronic Pancreatitis1 trial

Ivacaftor Impact in an Adolescent Without a Cystic Fibrosis-Associated CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) Mutation for Chronic Pancreatitis: Single-Patient Study Tracking Flare Frequency and Health Data Compared to Historical Records.

Active, not recruiting·MMission: Cure··NCT07022327·1 enrolled

ILD1 trial

(Study: Vertex IIS) Does Ivacaftor Alter Wild Type CFTR-Open Probability In The Sweat Gland Secretory Coil?

Completed·RRichard Barry Moss·N/A·NCT02310789·8 enrolled

Lung Disease1 trial

Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT02390219·46 enrolled

Chronic Bronchitis1 trial

VX-770 for the Treatment of Chronic Bronchitis

Withdrawn·UUniversity of Alabama at Birmingham·Phase 4·NCT03251911

Safety and Efficacy1 trial

The Topic Trial - Study to Determine the Safety and Efficacy of Ivacaftor

Completed·UUniversity of Alabama at Birmingham·Phase 2·NCT03085485·40 enrolled

PET1 trial

CPET in CF Patients With One G551D Mutation Taking VX770

Unknown status·TThe Alfred·Phase 4·NCT01937325·20 enrolled

Primary Ciliary Dyskinesia1 trial

Clearing Lungs With ENaC Inhibition in Primary Ciliary Dyskinesia

Completed·PParion Sciences·Phase 2·NCT02871778·123 enrolled

Efficacy and Safety1 trial

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 3·NCT02347657·510 enrolled

Cystic Fibrosis (CF)1 trial

A Low-intervention Prospective-retrospective Study to Evaluate the Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor Combination in a Cystic Fibrosis Population

Not yet recruiting·IIstituto Giannina Gaslini·Approved·NCT07809867·50 enrolled

Unspecified1 trial

Evaluation of Ivacaftor in Patients Using Ataluren for Nonsense Mutations

Completed·UUniversity of Alabama at Birmingham·Phase 4·NCT03256799·1 enrolled

Regulatory

FDA Approval Timeline[5]

Jan 31, 2012

KALYDECO

NDA NDA203188

May 3, 2023

KALYDECO

NDA NDA207925

Sources

References

[1]
ClinicalTrials.gov — 57 trial records for ivacaftor. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Kalydeco drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — ivacaftor compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for ivacaftor. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA207925). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Kalydeco. U.S. Food & Drug Administration.
[7]
SEC EDGAR — Vertex Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: Cystic Fibrosis Transmembrane Conductance Regulator Positive Modulator; Cystic Fibrosis Transmembrane Conductance Regulator Activator[3]
Modality: Small molecule[3]
Highest Phase: Approved[1]
Company: Vertex Pharmaceuticals