|ORKAMBI® Lumacaftor/Ivacaftor 200mg/125mg tablets
|The CFTR protein is a chloride channel present at the surface of epithelial cells in multiple organs. The F508del mutation impacts the CFTR protein in multiple ways, primarily by causing a defect in cellular processing and trafficking that reduces the quantity of CFTR at the cell surface. The small amount of F508del-CFTR that reaches the cell surface has low channel-open probability (defective channel gating). Lumacaftor is a CFTR corrector that acts directly on F508del-CFTR to improve its cellular processing and trafficking, thereby increasing the quantity of functional CFTR at the cell surface. Ivacaftor is a CFTR potentiator that facilitates increased chloride transport by potentiating the channel-open probability (or gating) of the CFTR protein at the cell surface. The combined effect of lumacaftor and ivacaftor is increased quantity and function of F508del-CFTR at the cell surface, resulting in increased chloride ion transport. The exact mechanisms by which lumacaftor improves cellular processing and trafficking of F508del-CFTR and ivacaftor potentiates F508del-CFTR are not known.
|Orkambi tablets are indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who are homozygous for the F508del mutation in the CFTR gene
|Orkambi 100 mg/125 mg film-coated tablets
Each film-coated tablet contains 100 mg of lumacaftor and 125 mg of ivacaftor.
Orkambi 200 mg/125 mg film-coated tablets
Each film-coated tablet contains 200 mg of lumacaftor and 125 mg of ivacaftor.