ORKAMBI (lumacaftor / ivacaftor), CFTR gene corrector and potentiator

High clinical benefit and minor clinical added valuein the management of cystic fibrosis, in patients aged between 6 and 11 years, homozygous for the F508del mutation of the CFTR gene. 

• ORKAMBI has now been granted a marketing authorisation for the treatment of cystic fibrosis in patients aged between 6 and 11 years, homozygous for the F508del mutation of the CFTR gene.

• The efficacy of ORKAMBI is based exclusively on one intermediate endpoint: improvement in lung clearance index assessed in the short-term (24 weeks) relative to placebo, with no demonstrated impact on quality of life.

• Its safety profile is similar to that observed in adolescents and adults.