Vertex cystic fibrosis drug Trikafta gets US approval

Vertex Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for its three-drug combination Trikafta to treat cystic fibrosis. The medicine is for use in patients aged 12 years and above with a minimum of one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Cystic fibrosis is a rare genetic disease caused by defective or missing CFTR protein, which forms from CFTR gene mutations. Of all CFTR mutations, at least one F508del mutation is known to be the most frequent in CF patients. Trikafta, which combines elexacaftor, tezacaftor and ivacaftor, boosts the F508del-CFTR protein quantity and function at the cell surface. The drug can be prescribed to around 6,000 patients with one F508del mutation and one minimal function mutation (F / MF), along with nearly 12,000 of those who have one or two F508del mutations and are eligible for one of the company’s three FDA-approved drugs for CF.