CHMP Recommends Marketing Authorization for Triple Combination Therapy for Cystic Fibrosis

July 1, 2020

EMA’s Committee for Human Medicinal Products (CHMP) has recommended that Kaftrio, a triple combination therapy for the treatment of cystic fibrosis, be authorized for marketing in the European Union.

The European Medicines Agency’s (EMA’s) Committee for Human Medicinal Products (CHMP) has recommended that Kaftrio, a triple combination therapy for the treatment of cystic fibrosis, be authorized for marketing in the European Union. The recommendation was announced in a June 26, 2020 press release.

Kaftrio, from Vertex Pharmaceuticals, is a therapy that combines three substances, elexacaftor, tezacaftor, and ivacaftor, which work together to increase the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The treatment is aimed at patients aged 12 years and older who are homozygous or heterozygous for the F508del mutation in the CFTR gene.

Safety and efficacy of the combination therapy have been studied in two clinical trials involving more than 500 patients. In both trials, a clinically significant improvement in lung function and a decrease of sweat chloride was found after treatment.

CHMP’s opinion will be submitted to the European Commission for a final decision on EU-wide marketing authorization.

Source: EMA