NDAORALGRANULEPriority Review
Approved
Aug 2018
Lifecycle
Peak
Competitive Pressure
8/100
Clinical Trials
7
Data completeness
6/8 — Strong
Priority Review
ORKAMBI (lumacaftor and ivacaftor) by Vertex Pharmaceuticals is chloride channel activation potentiators [moa]. Approved for cystic fibrosis transmembrane conductance regulator potentiator [epc]. First approved in 2018.
Drug data last refreshed 1h ago · AI intelligence enriched 3w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ORKAMBI (lumacaftor/ivacaftor) is a dual-action oral therapy for cystic fibrosis that combines a CFTR corrector and potentiator to improve protein folding and channel function. It treats CF patients with F508del-CFTR mutations by addressing the root cause of disease through cytochrome P450 3A induction. This combination approach represents a foundational shift toward precision medicine in rare genetic disease.
$0.001BPart D
Peak4.8 years to LOEProduct remains at peak revenue but facing significant competitive displacement from next-generation modulators; team consolidation and strategic transition planning likely underway.
Mechanism of Action
Chloride Channel Activation Potentiators
Pharmacologic Class:
Cystic Fibrosis Transmembrane Conductance Regulator Potentiator
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (7)
Orkambi Treatment in 2 to 5 Year Old Children With CF
Started Apr 2019
28 enrolled
Cystic Fibrosis
Gastrointestinal Study at Orkambi Therapy in CF Patients
Started Feb 2019
20 enrolled
Cystic Fibrosis
Safety and Pharmacokinetic Study of Lumacaftor/Ivacaftor in Participants 1 to Less Than 2 Years of Age With Cystic Fibrosis, Homozygous for F508del
Started Sep 2018
61 enrolled
Cystic Fibrosis
Impact of the Introduction of ORKAMBI on Anxiety, Depression, Quality of Life and Adherence of Adolescents and Young Adults
Started Dec 2017
Cystic Fibrosis
GLPG2737 on Top of Orkambi in Subjects With Cystic Fibrosis
Started Nov 2017
22 enrolled
Cystic Fibrosis
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.