– If approved, people ages 12 years and older who have one copy of the F508del mutation and a gating (F/G) or residual function (F/RF) mutation will now be eligible for triple combination therapy
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination with ivacaftor 150 mg tablets for the treatment of cystic fibrosis (CF) in all patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation worldwide. If the European Commission follows the recommendation, the majority of people with CF in Europe will be eligible for the medicine.
The CHMP positive opinion was based on the results from a global Phase 3 study (Study 445-104) evaluating the triple combination therapy in CF patients ages 12 years and older who are heterozygous for the F508del-CFTR mutation and a CFTR gating mutation (F/G) or a residual function mutation (F/RF). The study was conducted by Vertex to complement the prior Phase 3 trials, which showed positive results for ivacaftor / tezacaftor / elexacaftor in combination with ivacaftor in people ages 12 years and older with CF with two F508del mutations (F/F) or one F508del mutation and one minimal function mutation (F/MF) genotype. It showed statistically significant and clinically meaningful improvements in primary and key secondary endpoints, including lung function in patients treated with ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor.
“Today’s opinion is an important step towards bringing this medicine to any patient with at least one F508del mutation, including those with a gating or residual function mutation who were not previously eligible for the triple combination therapy,” said Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex.
In Europe, KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is currently licensed for the treatment of people with CF ages 12 years and older with an F/F or F/MF genotype.