ORKAMBI
ORKAMBI is a combination of lumacaftor and ivacaftor indicated for the treatment of cystic fibrosis in patients aged 1 year and older. The treatment is specifically for patients who are homozygous for the F508del mutation in the CFTR gene, which should be confirmed by an FDA-cleared mutation test if the genotype is unknown. The safety and efficacy of the drug have not been established in cystic fibrosis patients who do not carry this specific homozygous mutation.
How ORKAMBI Works
The F508del mutation causes CFTR protein misfolding and degradation, leading to reduced protein quantity and defective channel gating at the cell surface. Lumacaftor improves the conformational stability of the F508del-CFTR protein, which increases the processing and trafficking of mature protein to the cell surface. Ivacaftor functions as a CFTR potentiator that increases chloride transport by improving the channel-open probability, or gating, of the protein. Together, these components increase the quantity, stability, and function of the CFTR protein to enhance chloride ion transport.
Details
- Status
- Prescription
- First Approved
- 2015-07-02
- Routes
- ORAL
- Dosage Forms
- TABLET, GRANULE
ORKAMBI Approval History
What ORKAMBI Treats
1 indicationsORKAMBI is approved for 1 conditions since its original approval in 2015. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Cystic Fibrosis
Drugs Similar to ORKAMBI
FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
ORKAMBI FDA Label Details
ProIndications & Usage
FDA Label (PDF)ORKAMBI is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene. If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene. ORKAMBI is a combination of ivacaftor, a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, and lumacaftor, indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene...
ORKAMBI Patents & Exclusivity
Patents (566 active)
Exclusivity
Want competitive intelligence?
See who's developing similar drugs and track their progress
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.