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lumacaftor

Phase 2Small molecule

Vertex Pharmaceuticals

lumacaftor is one of 4 small molecule programs targeting Heart Failure With Reduced Ejection Fraction. It is currently in Phase 2.

1

Indications

11

Total Trials

1

Active Trials

3

Competitors

Pipeline

Status by Indication[3][1]

Indication

Heart Failure With Reduced Ejection FractionRecruiting

Heart Failure With Reduced Ejection FractionPhase 2

Recruiting

Competition

Competitive Landscape

4 competitors in Heart Failure With Reduced Ejection Fraction

#DrugCompanyPhaseTrials

Hfref Polypill

WWashington University School of Medicine

Proamatine

Ozempic

lumacaftor

Vertex Pharmaceuticals

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

Timeline

Expected Milestones

Q3 2027

Phase 2Recruiting

Heart Failure With Reduced Ejection Fraction

NCT07695090

Clinical

Trials by Indication[1]

Cystic Fibrosis5 trials

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 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

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

Show 2 more trials

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 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

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

Adult Subject1 trial

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

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

Moderate Hepatic Impairment1 trial

Study of Lumacaftor in Combination With Ivacaftor in Subjects With Moderate Hepatic Impairment and Healthy Subjects

Completed· Vertex Pharmaceuticals logo

Vertex Pharmaceuticals·Phase 1·NCT01888393·23 enrolled

1 more indication

Heart Failure With Reduced Ejection Fraction1 trial

A Randomized, Parallel Group, Placebo-controlled, Double-blind, Longitudinal, Single Treatment Center, Phase II Proof-of-concept Study to Evaluate the Efficacy and Safety of Lumacaftor in Stable Heart Failure Subjects With Reduced Ejection Fraction

Recruiting·QQanatpharma AG·Phase 2·NCT07695090·60 enrolled

Sources

References

[1]
ClinicalTrials.gov — 11 trial records for lumacaftor. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — lumacaftor compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for lumacaftor. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Vertex Pharmaceuticals company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: Vertex Pharmaceuticals

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