Avalglucosidase alfa is in clinical development for the treatment of late-onset Pompe disease. Pompe disease is an inherited, genetic disorder which results in the deficiency of the enzyme ‘acid alpha-glucosidase’ (GAA). This deficiency leads to progressive accumulation of glycogen, a type of sugar, usually stored in muscle tissues particularly around the heart, skeletal muscle and respiratory muscles. Late-onset Pompe disease develop after one year of age, and is a serious, progressive, debilitating, and ultimately life threatening disease associated with high morbidity. Enzyme replacement therapy with alglucosidase alfa is a recommended treatment approach but the main drawback of the current option is the limited uptake by affected muscles leading to limited clinical benefits in some patients.
Avalglucosidase alfa is a next-generation enzyme replacement therapy that works by providing GAA enzyme activity in patients with Pompe disease. It is a chemically modified version of alglucosidase alfa that is designed for improved delivery to affected muscles with a potential for improving muscle coordination, strength, and respiratory function of patients with Pompe disease. Early studies found that avalglucosidase alfa improved symptoms in both patients who have previously received alglucosidase alfa and those who have not. If licensed, avalglucosidase alfa will offer an additional treatment option for late-onset Pompe disease.
Daratumumab in addition to cyclophosphamide, bortezomib and dexamethasone (CyBorD) is in clinical development for newly diagnosed systemic amyloid light-chain (AL) amyloidosis in adults. AL amyloidosis belongs to a group of diseases called systemic amyloidosis in which deposits of proteins (called amyloids) accumulate and cause damage in tissues and organs such as the kidneys, liver, gut, heart and nerves. In AL amyloidosis, the deposits are made up of proteins (called immunoglobulin light chains) produced in excess by malfunctioning white blood cells in the bone marrow.