KALYDECO (ivacaftor), selective potentiator of the CFTR protein

TRANSPARENCY COMMITTEE OPINION

Opinions on drugs - Posted on Oct 13 2016

Inclusion

KALYDECO 50 and 75 mg has Marketing Authorisation in children aged 2 years and older with cystic fibrosis, weighing less than 25 kg, who have one of the following CFTR gene regulation defect mutations (class III): G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.
Its efficacy was demonstrated in children aged 6 years and older, who have these mutations, in particular in terms of short- and medium-term improvement of FEV1. The consequences of these results on the evolution of the patients are unknown, but it seems that established pulmonary lesions cannot heal. Based on the lack of data on long-term morbidity and mortality, the benefit of the medicinal product in the overall management of the disease and its evolution remain to be shown.
The safety profile in children and in adolescents is comparable to that seen in adults.