Therapy Areas: Hereditary Disorders
US FDA grants approval to Vertex Pharmaceuticals' TRIKAFTA
23 October 2019 -

The United States Food and Drug Administration (US FDA) has granted approval to United States-based Vertex Pharmaceuticals for its TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) intended 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, it was reported yesterday.

This approval will allow around 6,000 people with CF aged 12 years and older who have one F508del mutation and one minimal function mutation to have a medicine that targets the underlying cause of their CF. In addition to that, around 12,000 people with one or two F508del mutations who are presently eligible for one of Vertex's three other US FDA-approved CF medicines are now also eligible for TRIKAFTA.

The company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the elexacaftor/tezacaftor/ivacaftor combination regimen. Presently, the company is assessing elexacaftor/tezacaftor/ivacaftor in people aged six to 11 with F/MF and F/F CF mutations in an ongoing Phase three study and is committed to assessing elexacaftor/tezacaftor/ivacaftor in children greater than six years of age as part of planned future studies.