Friday, July 10, 2026
🇺🇸 FDA

Drug profile · INN

lumacaftor

ORKAMBI

lumacaftor (ORKAMBI) is a cystic fibrosis transmembrane conductance regulator stabiliser. Associated with Vertex Pharmaceuticals (Australia) Pty..

EMA: approved Respiratory system (R07) R07AX30
Patents linked 20
Pipeline programs 4
News articles 0
Drug details — lumacaftor
Class
Respiratory system (R07)
Route
—
Patents
20
Programs
4
Data quality
0.89
← All drugs Manufacturer profile Related patents Pipeline programs Drug news hub Event calendar

Quick answer

lumacaftor (ORKAMBI) is a cystic fibrosis transmembrane conductance regulator stabiliser. Associated with Vertex Pharmaceuticals (Australia) Pty..

Key facts

INN lumacaftor
Brand ORKAMBI
Mechanism Cystic fibrosis transmembrane conductance regulator stabiliser
Target Cystic fibrosis transmembrane conductance regulator
Therapeutic class Respiratory system (R07)
ATC code R07AX30
Originator Vertex Pharmaceuticals (Australia) Pty.

Mechanism of action

Cystic fibrosis transmembrane conductance regulator stabiliser

Primary target: Cystic fibrosis transmembrane conductance regulator

Approval history

Authority Status Date
EMA approved —

Pipeline programs

Search pipeline Sponsor programs
  1. Lumacaftor Phase 3 · Vertex Pharmaceuticals (Australia) · Cystic Fibrosis
  2. Lumacaftor Plus Ivacaftor Combination Phase 3 · Vertex Pharmaceuticals (Australia) · Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
  3. Lumacaftor Plus Ivacaftor Combination Phase 3 · Vertex Pharmaceuticals (Australia) · Cystic Fibrosis, Homozygous or Heterozygous for the F508del-
  4. lumacaftor 200 mg q12h + ivacaftor 250 mg q12h Phase 1 · Vertex Pharmaceuticals (Australia) · Hepatic Impairment

Related & competitor drugs

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

Related patents

Browse patents Vertex Pharmaceuticals (Australia) Pty. patent hub

  1. 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

  2. US 11564916

    Pharmaceutical composition and administrations thereof

    Expires Mon Jan 26 2043 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  3. US 11052075

    Pharmaceutical compositions of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl) benzoic acid and administration thereof

    Expires Mon Jul 01 2041 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  4. US 10646481

    Pharmaceutical composition and administrations thereof

    Expires Mon May 07 2040 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  5. US 10597384

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

    Expires Mon Mar 19 2040 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  6. US 10076513

    Pharmaceutical compositions of 3-(6-(1-(2,2-difluorobenzo[D][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl) benzoic acid and administration thereof

    Expires Mon Sep 13 2038 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  7. US 9931334

    Solid forms of N[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide

    Expires Mon Mar 29 2038 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  8. US 9670163

    Solid forms of N-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide

    Expires Mon Jun 01 2037 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  9. US 9216969

    Modulators of ATP-binding cassette transporters

    Expires Mon Dec 17 2035 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  10. US 9192606

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

    Expires Mon Nov 19 2035 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  11. US 9150552

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

    Expires Mon Oct 01 2035 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  12. US 8993600

    Modulators of ATP-binding cassette transporters

    Expires Mon Mar 26 2035 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  13. US 8846718

    Solid forms of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxo1-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl) benzoic acid

    Expires Mon Sep 25 2034 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  14. US 8754224

    Solid forms of N-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide

    Expires Mon Jun 12 2034 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  15. US 8741933

    Modulators of ATP-binding cassette transporters

    Expires Mon May 29 2034 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  16. US 8716338

    Dosage units of 3-(6-(1-(2,2-difluorobenzo[D] [1,3] dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid

    Expires Mon May 01 2034 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  17. US 8653103

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

    Expires Mon Feb 13 2034 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  18. US 8507534

    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 08 2033 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  19. US 8410274

    Solid forms of N-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide

    Expires Mon Mar 28 2033 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

  20. US 8324242

    Modulators of ATP-binding cassette transporters

    Expires Mon Nov 29 2032 00:00:00 GMT+0000 (Coordinated Universal Time)

    granted

Frequently asked questions

What is lumacaftor?

lumacaftor (ORKAMBI) is a cystic fibrosis transmembrane conductance regulator stabiliser. Associated with Vertex Pharmaceuticals (Australia) Pty..

What is the brand name for lumacaftor?

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

Drug approvals, clinical trials, M&A, event intelligence, and company updates — weekly.

Customize topics & region
Topics
Region

No spam. Unsubscribe anytime. See our privacy policy.