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 14h ago · AI intelligence enriched 3w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ORKAMBI is a fixed-dose combination of lumacaftor and ivacaftor approved in 2018 for cystic fibrosis patients with specific CFTR mutations. It works by activating chloride channels in the CFTR protein, restoring ion transport and reducing mucus buildup in the lungs. Administered as an oral granule, it addresses the underlying cause of CF rather than just symptoms.
$0.001BPart D
Peak4.6 years to LOEPeak lifecycle stage indicates stable commercial execution with established market presence; team focus is on patient persistence and market maintenance rather than rapid expansion.
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.