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

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.

Gefapixant is an oral drug that blocks receptors in the sensory cells in the airways and lungs, potentially reducing cough frequency in patients with refractory or unexplained chronic cough. Preliminary results from early studies have demonstrated that gefapixant is efficacious and safe. If licenced, gefapixant could provide a treatment option for patients with refractory or unexplained cough who have no licensed therapies available and few effective therapies available.

Vericiguat is a medicinal product in clinical development for the treatment of heart failure (HF) with reduced ejection fraction. HF is a complex clinical syndrome of symptoms and signs that suggest the efficiency of the heart as a pump is impaired. Symptoms include breathlessness and fatigue, and signs of the condition include swollen ankles and crackling sounds in the lungs. More than half of people with HF have a reduced ejection fraction (HFrEF), also referred to as systolic heart failure, where the heart muscle does not contract effectively, and therefore less oxygen-rich blood is pumped out to the body. There remains a large unmet need for new therapies in the treatment of HFrEF.