The EMA’s human medicines committee (CHMP) has recommended granting a marketing authorisation in the EU for Kaftrio.
Kaftrio is the first triple combination therapy for the treatment of cystic fibrosis (CF) in patients 12 years and older, who are homozygous for the F508del mutation, or heterozygous for F508del in the cystic fibrosis transmembrane conductance regulator (CFTR) gene with minimal function mutation. These two groups represent the majority of patients diagnosed with CF.
Kaftrio is made up of elexacaftor, tezacaftor, and ivacaftor, which increase the CFTR protein and improve its function. It is the first treatment that is effective for CF patients with the F508del mutation on one allele and an MF mutation on the second allele.
Safety and efficacy of Kaftrio were studied in two clinical trials, involving over 500 patients. Patients heterozygous for F508del with an MF mutation and patients homozygous for F508del mutation had clinically significant improvements in their lung function by 14.3 and 10 percentage points, respectively.
The opinion adopted by the CHMP is an intermediary step on Kaftrio’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation.