Vertex Pharmaceuticals Incorporated (Canada) announced that it has signed a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA), which represents an agreement in principle regarding the public reimbursement of PrTRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for eligible patients with cystic fibrosis (CF). This is an extension of the LOI with the pCPA including PrKALYDECO® (ivacaftor) and PrORKAMBI® (lumacaftor/ivacaftor). This extension of the LOI follows the positive clinical recommendation for TRIKAFTA® by both the Canadian Agency for Drugs and Technology in Health (CADTH) and l'Institut national d'excellence en santé et en services sociaux (INESSS) in Quebec. TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. TRIKAFTA® is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. The approval of TRIKAFTA® was supported by positive results of three global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study (Study 445-102) in 403 people with one F508del mutation and one minimal function mutation (F/MF), a four-week Phase 3 study (Study 445-103) in 107 people with two F508del mutations (F/F), and a Phase 3 study (Study 445-104) in 258 people heterozygous for the F508del-CFTR mutation and a CFTR gating mutation (F/G) or a residual function mutation (F/RF). Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 80,000 people globally. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.