Inclisiran is in clinical development for treatment of hypercholesterolaemia in patients at high risk of cardiovascular disease in addition to, or after use of, oral anti‐lipidaemics
including statins. Abnormal levels of lipids in the blood characterises dyslipidaemia. High levels of cholesterol in the blood (hypercholesterolemia) may be caused by inherited genetic
defects as seen in familial hypercholesterolaemia, or may occur when genes and other factors such as lifestyle habits interact, as seen in non‐familial hypercholesterolaemia.
Elevated levels of low‐density lipoprotein cholesterol (LDL‐C) increase the risk of cardiovascular disease, which is responsible for many deaths and disabilities.
The current standard of care for patients with hypercholesterolaemia is primarily statins which are capable of reducing LDL‐C. There is however a subset of patients who are unable
to reach LDL‐C goals despite maximally tolerated oral lipid lowering therapy. Inclisiran is a medicinal product that inhibits a protein called PCSK9 especially present on the liver cells
leading to a decrease in circulating LDL‐C. If licensed, inclisiran will offer an additional treatment option for patients with primary hypercholesterolaemia as adjunctive therapy to diet and in combination with other lipid‐lowering therapies.
Treosulfan in addition to fludarabine is in clinical development for paediatric non-malignant disease prior to allogeneic stem cell transplant. Treosulfan is a medicine given to patients before they have a bone marrow transplant from a donor known as ‘allogeneic haematopoietic stem cell transplantation’. It is used as a ‘conditioning’ treatment to clear the patient’s bone marrow and make room for the transplanted bone marrow cells, which can then produce healthy blood cells. Treosulfan is used together with another medicine called fludarabine for the treatment of a variety of disorders that require a bone marrow transplant.