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.
Nitisinone is in clinical development for the treatment of alkaptonuria. Alkaptonuria is a rare metabolic disorder, in which patients lack a functional enzyme that prevents the body fully breaking down two amino acids called tyrosine and phenylalanine. This results in a build-up of a chemical called homogentisic acid (HGA) in the body, which is deposited as black pigment in tissues, in a process called ochronosis. This results in dark colouration of urine, joint problems, breathing difficulties and heart, kidney and prostate problems. In the later stage, patients may experience physical disability and inability to perform daily activities. Early recognition and management of alkaptonuria is desirable to slow the progression of this disease.