NDAORALTABLETPriority Review
Approved
Jul 2015
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 2015.
Drug data last refreshed 17h ago · AI intelligence enriched 2w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ORKAMBI is an oral combination therapy containing lumacaftor and ivacaftor, approved by the FDA in 2015 for cystic fibrosis (CF). It targets the cystic fibrosis transmembrane conductance regulator (CFTR) by combining a corrector (lumacaftor) that helps misfolded CFTR protein reach the cell surface with a potentiator (ivacaftor) that enhances chloride channel function. This dual mechanism addresses the underlying defect in homozygous F508del-CFTR mutations.
$0.001BPart D
Peak4.6 years to LOEProduct is at peak lifecycle stage with relatively modest Part D spending ($1M in 2023), suggesting mature but challenged market penetration within the CF patient population.
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
Ivacaftor Treatment in 4 Month to 2 Year Old CF Subjects
Started Feb 2019
15 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.