Vertex Pharmaceuticals Incorporated declared that the European Commission has conceded approval of the label extension for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in all patients ages 12 years and older who have at any rate one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. With this extension of the indication, CF patients 12 years and older who are heterozygous for the F508del-CFTR mutation and a residual function (F/RF) or gating mutation (F/G) are eligible for the triple combination therapy interestingly, and most of individuals ages 12 years and older with CF in Europe are presently eligible for KAFTRIO.
Vertex KAFTRIO® Will Help CF Patients
“This indication extension is significant as CF patients in Europe with gating and residual function mutations will approach KAFTRIO interestingly,” said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. “We anticipate working with health authorities and governments to guarantee that all eligible patients who can profit by this medicine approach straightaway.” In both the United States and Australia, where the triple combination therapy is known as TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), it is now licensed for the treatment of individuals with CF ages 12 years and older who have at any rate one copy of the F508del mutation in the CFTR gene, paying little heed to the next mutation type.
“In clinical trials ivacaftor/tezacaftor/elexacaftor in addition to ivacaftor showed positive critical outcomes in individuals with cystic fibrosis ages 12 years and older who have at any rate one copy of the most common gene mutation (F508del) with the most recent outcomes showing clinical advantages in people who have an extra ‘gating’ (F/G) or ‘residual function’ (F/RF) gene mutation. It is welcome information for the CF people group that the European regulatory authority has broadened the licensed indications for this therapy dependent on the most recent preliminary outcomes which implies that extra patients will access this medicine,” said Dr Peter Barry, Honorary Senior Lecturer at The University of Manchester.
Because of long haul reimbursement agreements in the Republic of Ireland, Northern Ireland, Denmark and Luxembourg, and provisions for access in health care systems like Germany, eligible patients in these nations will approach the extended indication for the triple combination regimen in no time following regulatory approval by the European Commission.