VX-659/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 12 years and older

Icosapent ethyl is in clinical development as a treatment to reduce the risk of cardiovascular events in high-risk patients who have their cholesterol levels controlled with statin treatment, but have elevated triglycerides and other cardiovascular risk factors. Cardiovascular events include heart attack, angina and stroke. These diseases are the main cause of death in the UK, accounting for over a quarter of deaths each year. Patients receiving statin treatment are still at a high risk and would benefit from treatment to reduce cardiovascular events.

Empagliflozin is given by mouth in the form of a tablet and works by inhibiting the sodium transporter NHE1. This prevents salt from being re-absorbed so there is increased excretion of salt from the body and a reduced volume of fluid in the blood vessels. It’s thought that these changes in sugar, salt and water metabolism in the body may contribute to the reductions in cardiovascular death. If licensed, empagliflozin may provide an additional treatment option for people with HFrEF who currently have limited therapies available.

Elexacaftor and tezacaftor are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. The triple therapy of elexacaftor/tezacaftor/ivacaftor-FDC may result in an effective therapeutic option for people with CF heterozygous for F508del mutations, who currently have limited options.