lumacaftor and ivacaftor

FDA Drug Profile — ORKAMBI

lumacaftor and ivacaftor is listed in the FDA National Drug Code directory with 5 NDC codes, marketed under brand name including ORKAMBI. Available dosage forms: granule, tablet, film coated.

Drug Details

Generic Name
lumacaftor and ivacaftor
Brand Names
ORKAMBI
Application Number
NDA211358
Sponsor
Vertex Pharmaceuticals Incorporated
NDC Codes
5
Dosage Forms
GRANULE, TABLET, FILM COATED
Routes
ORAL
Active Ingredients
LUMACAFTOR, IVACAFTOR

Indications and Usage

1 INDICATIONS AND USAGE 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. 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. ( 1 ) Limitations of Use: The efficacy and safety of ORKAMBI have not been established in patients with CF other than those homozygous for the F508del mutation. ( 1 ) Limitations of Use The efficacy and safety of ORKAMBI have not been established in patients with CF other than those homozygous for the F508del mutation .