Lumacaftor/ivacaftor

Lumacaftor/ivacaftor

Combination of
Lumacaftor	CFTR potentiator
Ivacaftor	CFTR potentiator
Clinical data
Trade names	Orkambi
AHFS/Drugs.com	entry
Pregnancy category	US: B (No risk in non-human studies)
Legal status	US: ℞-only
Routes of administration	Oral
Identifiers
ATC code	R07AX30

Lumacaftor/ivacaftor (brand name Orkambi) is a drug used for the treatment of cystic fibrosis. It is a combination drug that consists of lumacaftor and ivacaftor. In July 2015, the Food and Drug Administration (FDA) approved the drug for people with cystic fibrosis with the F508del mutation and an age of 12 years or older.^[1] The approval of the drug was based on clinical trial data.^[2][3][4]

Ivacaftor is considered a CFTR potentiator (increases activity of CFTR protein at the surface of epithelial cell), while lumacaftor is a CFTR corrector (increases trafficking of CFTR protein to the cell surface). The drug has received breakthrough therapy designation from the FDA.

In Denmark, it is estimated that if the drug is introduced in the treatment the cost will amount to 2 million Danish krones (approximately 270,000 euro) each year per person.^[5] The cost in the United States is approximately $259,000 per person per year. It is sold by Vertex Pharmaceuticals Incorporated.

References