Posted by Michael Wonder on 30 Mar 2022
Cystic fibrosis: early access granted to the Kaftrio/Kalydeco combination
29 March 2022 - The High Authority for Health has authorised early access to a first treatment for children aged 6 to 11, but only those who are heterozygous for the F508del mutation of the CFTR gene and carriers of a mutation of this same gene with minimal function.
This is the combination of Kaftrio (ivacaftor with tezacaftor and elexacaftor) and Kalydeco (ivacaftor), which the HAS Transparency Commission has already evaluated positively for children aged 12 and over.
