August 2019
Elexacaftor/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation and one minimal function mutation in patients aged 6 to 11 years
The triple fixed-dose combination (FDC), elexacaftor/tezacaftor/ivacaftor-FDC, is in clinical development for cystic fibrosis (CF) that is heterozygous for F508del mutation and a minimal function mutation for patients aged 6 to 11 years old. CF is the most common, life-limiting recessively inherited (a faulty gene inherited from both parents) disease in the UK. Genetic mutations affect the CF transmembrane conductance regulator (CFTR) gene, which is essential for the regulation of salt and water movements across cell membranes. These mutations mean that the CFTR protein is not processed and moved through the cells normally, resulting in little to no CFTR protein at the cell surface. This results in thickened secretions in organs with epithelial cell lining, mainly affecting the lungs and digestive system.
August 2019
Elexacaftor/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 6 to 11 years old
The triple fixed-dose combination (FDC), elexacaftor/tezacaftor/ivacaftor-FDC, is in clinical development for cystic fibrosis (CF) that is homozygous for F508del mutation for patients aged 6 to 11 years old. CF is the most common, life-limiting recessively inherited (a faulty gene inherited from both parents) disease in the UK. Genetic mutations affect the CF transmembrane conductance regulator (CFTR) gene, which is essential for the regulation of salt and water movements across cell membranes. These mutations mean that the CFTR protein is not processed and moved through the cells normally, resulting in little to no CFTR protein at the cell surface. This results in thickened secretions in organs with epithelial cell lining, mainly affecting the lungs and digestive system.
August 2019
Ticagrelor in addition to acetylsalicylic acid for prevention of stroke in patients with acute ischaemic stroke or transient ischaemic attack
Ticagrelor in addition to acetylsalicylic acid (ASA) is in development for the prevention of new stroke in patients with acute ischaemic stroke or high-risk transient ischaemic attack. A stroke is a serious life-threatening medical condition that happens when the blood supply to part of the brain is cut off. There are three different types of stroke; ischaemic strokes, haemorrhagic strokes and transient ischaemic attacks. The aim of stroke therapy is to restore optimal blood flow to the brain, reduce any damage caused to the brain tissues, modulate any factors that may exacerbate this damage and if possible, repair the damage. Early treatment is critical to rescue potentially salvageable brain tissue.
June 2019
Lumacafor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 12 to 23 months
The fixed-dose combination (FDC) lumacaftor/ivacaftor-FDC is in clinical development for cystic fibrosis (CF) that is homozygous for F508del mutation for patients aged 12 to 23 months. CF is the most common, life-limiting recessively inherited (a faulty gene inherited from both parents) disease in the UK. Genetic mutations affect the CF transmembrane conductance regulator (CFTR) gene, which is essential for the regulation of salt and water movements across cell membranes. These mutations mean that the CFTR protein is not processed and moved through the cells normally, resulting in little to no CFTR protein at the cell surface. This results in thickened secretions in organs with epithelial cell lining, mainly affecting the lungs and digestive system.
