The European Medicines Agency’s Committee for Medicinal Products for Human Use has recommended granting a conditional marketing authorisation across the EU for Vertex’s triple therapy Kaftrio (elexacaftor/tezacaftor/ivacaftor) in combination with Kalydeco (ivacaftor) for cystic fibrosis patients 12 years and older with either one F508del mutation and one minimal function mutation or two F508del mutations in the CFTR (cystic fibrosis transmembrane conductance regulator) gene. This opinion came on the back of results from the AURORA F/MF (NCT03525444) and AURORA F/F (NCT03525548) phase III clinical trials where the triple combination significantly improved lung function and reduced pulmonary exacerbations in such CF patients. Kaftrio is already approved and marketed in the U.S. under the name Trikafta, and this CHMP opinion will go to the EC for a final decision.
Marcus A. Mall, MD, professor at Charité University Medical Center Berlin, said: “The clinical data for ivacaftor/tezacaftor/elexacaftor plus ivacaftor in people with CF ages 12 years and older with an F/F or F/MF genotype are unprecedented. In addition to improvements in lung function, the data have shown improvements in multiple important outcome measures, including quality of life. Both the clinical and patient communities are excited that more people with CF will be able to benefit from CFTR modulators.”
“ If approved, this would be the first CFTR modulator for people with one F508del mutation and one minimal function mutation and would bring additional benefit to people with two F508del mutations“
Carmen Bozic, MD, executive vice president of global medicines development and medical affairs, and chief medical officer at Vertex Pharmaceuticals, said: “We are delighted to have received this positive opinion from CHMP. If approved, this would be the first CFTR modulator for people with one F508del mutation and one minimal function mutation and would bring additional benefit to people with two F508del mutations. This milestone brings us one step closer to delivering this innovative CF medicine to those who are waiting, and toward our ultimate goal of providing a therapeutic option for every person with this rare and devastating disease.”