Wednesday, July 8, 2026

Drug profile · INN

ivacaftor

ivacaftor (KALYDECO) is a cystic fibrosis transmembrane conductance regulator positive modulator. Associated with Vertex Pharmaceuticals (Australia) Pty.. Route of administration: oral. Current US regulatory status: approved.

FDA: approved EMA: approved TGA: approved Respiratory system (R07) R07AX32
US status approved
Patents linked 20
Pipeline programs 18
News articles 1
Drug details — ivacaftor

Quick answer

ivacaftor (KALYDECO) is a cystic fibrosis transmembrane conductance regulator positive modulator. Associated with Vertex Pharmaceuticals (Australia) Pty.. Route of administration: oral. Current US regulatory status: approved.

Key facts

INN ivacaftor
Brand KALYDECO
Mechanism Cystic fibrosis transmembrane conductance regulator positive modulator
Target Cystic fibrosis transmembrane conductance regulator
Therapeutic class Respiratory system (R07)
Route ORAL
Formulation GRANULE
ATC code R07AX32

Mechanism of action

Cystic fibrosis transmembrane conductance regulator positive modulator

Primary target: Cystic fibrosis transmembrane conductance regulator

Approval history

Authority Status Date
FDA approved
EMA approved
TGA approved

Recent coverage

Pipeline programs

  1. Ivacaftor Phase 3 · Vertex Pharmaceuticals (Australia) · Cystic Fibrosis
  2. Lumacaftor Plus Ivacaftor Combination Phase 3 · Vertex Pharmaceuticals (Australia) · Cystic Fibrosis, Homozygous or Heterozygous for the F508del-
  3. Lumacaftor Plus Ivacaftor Combination Phase 3 · Vertex Pharmaceuticals (Australia) · Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
  4. Tezacaftor/Ivacaftor Phase 3 · Vertex Pharmaceuticals (Australia) · Cystic Fibrosis
  5. VX-661 Plus Ivacaftor Combination Phase 3 · Vertex Pharmaceuticals (Australia) · Cystic Fibrosis
  6. VX-661/Ivacaftor Phase 3 · Vertex Pharmaceuticals (Australia) · Cystic Fibrosis
  7. VX20-445-112 Phase 3 · Vertex Pharmaceuticals (Australia) · Cystic Fibrosis
  8. VX21-121-105 Phase 3 · Vertex Pharmaceuticals (Australia) · Cystic Fibrosis

Related & competitor drugs

  1. desloratadine Respiratory system (R01)
  2. deutivacaftor Cystic fibrosis transmembrane conductance regulator activator
  3. deutivacaftor / tezacaftor / vanzacaftor Respiratory system (R07)
  4. lumacaftor Respiratory system (R07)
  5. mannitol Respiratory system (R05)
  6. reslizumab Respiratory system (R03)
  1. US 12421251

    Cystic fibrosis transmembrane conductance regulator modulating agents

    Expires Mon Sep 18 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  2. US 12384790

    Pteridinone compounds and uses thereof

    Expires Mon Aug 07 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  3. US 12377252

    Implantation devices, system, and methods

    Expires Mon Jul 31 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  4. US 12350262

    Methods of treatment for cystic fibrosis

    Expires Mon Jul 03 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  5. US 12324802

    Modulators of cystic fibrosis transmembrane conductance regulator

    Expires Mon Jun 05 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  6. US 12281057

    Carboxamides as modulators of sodium channels

    Expires Mon Apr 17 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  7. US 12269831

    Modulators of cystic fibrosis transmembrane conductance regulator

    Expires Mon Apr 03 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  8. US 12258333

    N-(hydroxyalkyl (hetero)aryl) tetrahydrofuran carboxamides as modulators of sodium channels

    Expires Mon Mar 20 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  9. US 12247021

    Substituted tetrahydrofurans as modulators of sodium channels

    Expires Mon Mar 06 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  10. US 12214083

    Pharmaceutical composition and administrations thereof

    Expires Mon Jan 30 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  11. US 12194138

    Devices and methods for delivering therapeutics

    Expires Mon Jan 09 2045 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  12. US 12168009

    Modulators of cystic fibrosis transmembrane conductance regulator

    Expires Mon Dec 12 2044 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  13. US 12065432

    Solid forms of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl) benzoic acid

    Expires Mon Aug 15 2044 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  14. US 12031150

    Methods, compositions and kits for increasing genome editing efficiency

    Expires Mon Jul 04 2044 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  15. US 11951212

    Pharmaceutical compositions for the treatment of cystic fibrosis transmembrane conductance regulator mediated diseases

    Expires Mon Apr 04 2044 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  16. US 11951206

    Cell housing device

    Expires Mon Apr 04 2044 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  17. US 11919887

    Substituted tetrahydrofurans as modulators of sodium channels

    Expires Mon Feb 29 2044 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  18. US 11827627

    N-(hydroxyalkyl (hetero)aryl) tetrahydrofuran carboxamides as modulators of sodium channels

    Expires Mon Nov 23 2043 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  19. US 11773119

    Prodrugs of pyridone amides useful as modulators of sodium channels

    Expires Mon Sep 28 2043 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted
  20. US 11752106

    Pharmaceutical composition and administrations thereof

    Expires Mon Sep 07 2043 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

Frequently asked questions

What is ivacaftor?

ivacaftor (KALYDECO) is a cystic fibrosis transmembrane conductance regulator positive modulator. Associated with Vertex Pharmaceuticals (Australia) Pty.. Route of administration: oral. Current US regulatory status: approved.

What is the brand name for ivacaftor?

KALYDECO is a marketed brand name for ivacaftor. Check FDA Drugs@FDA for the current US label holder and prescribing information.

What is the US approval status of ivacaftor?

ivacaftor has approved status in the United States according to our Drugs@FDA-derived records. Verify on the official FDA label before clinical or commercial decisions.