FDA approves Trikafta combination to treat cystic fibrosis in people who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. - Vertex Pharma

Vertex Pharmaceuticals Incorporated announced the FDA has approved Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in people 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. With this approval, for the first time, approximately 6,000 people with CF ages 12 years and older who have one F508del mutation and one minimal function mutation (F/MF) have a medicine that targets the underlying cause of their CF.

Additionally, approximately 12,000 people with one or two F508del mutations who are currently eligible for one of Vertex�s three other FDA-approved CF medicines are now also eligible for Trikafta.

Vertex has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the elexacaftor/tezacaftor/ivacaftor combination regimen. Vertex is currently evaluating elexacaftor/tezacaftor/ivacaftor in people ages 6 through 11 with F/MF and F/F CF mutations in an ongoing Phase III study and is committed to evaluating elexacaftor/tezacaftor/ivacaftor in children <6 years of age as part of planned future studies.>

Comment: Trikafta is now the first triple combination therapy on the market for patients 12 years and older with the most common cystic fibrosis mutation. That group represents an estimated 90% of the cystic fibrosis population, including 27,000 people in the U.S., according to the FDA.