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.
Tralesinidase alfa is an investigational enzyme replacement therapy (ERT) designed to replace the faulty enzyme with a healthy one in patients with MPS IIIB. Tralesinidase alfa is delivered directly to the fluid surrounding the brain (cerebrospinal fluid). If licensed, tralesinidase alfa will offer new therapy option for patients with MPS IIIB who currently have no treatment options.