Cardiovascular disease (CVD) is a general term that refers to all conditions affecting the heart and blood vessels (circulation). These include angina, heart attack (also known as myocardial infarction), heart failure, stroke, and a number of diseases that affect the blood vessels.
CVD is usually associated with a build-up of fatty deposits inside the blood vessels (arteries) which make the vessels become narrowed (atherosclerosis). The body also reacts to the fatty deposits by sending white blood cells to the blood vessels; this process is called inflammation. The fatty deposits along with the inflammation lead to reduction of the blood supply to the heart and significantly increase the risks of heart attack and other types of cardiovascular diseases.
Canakinumab is a medicine being developed to target inflammation. It is the first and only agent which has shown that by selectively targeting some specific biomarkers of inflammation, it significantly reduces cardiovascular risks. Canakinumab is currently being developed to be used as an add-on therapy for patients who have had a prior heart attack and have a higher risk of further cardiovascular disease.
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.