New triple therapy for cystic fibrosis

Country

United States

A new triple combination therapy for cystic fibrosis, targeting the most common genetic mutation behind the disease, has been approved by the US Food and Drug Administration. Trikafta (elexacaftor/ivacaftor/tezacaftor) is now available to patients 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This is said to represent about 90% of all patients with the disease.