bucelipase alfa

Bucelipase alfa is a recombinant human bile salt-stimulated lipase (rhBSSL) developed by Swedish Orphan Biovitrum (Sobi) for the treatment of exocrine pancreatic insufficiency (EPI), particularly in patients with cystic fibrosis and preterm infants. Bile salt-stimulated lipase is a versatile enzyme naturally secreted by the pancreas and present in breast milk, which requires bile salts for activation in the small intestine. It plays a critical role in the hydrolysis of a wide range of dietary lipids, including triglycerides, cholesteryl esters, and fat-soluble vitamins. As an enzyme replacement therapy, bucelipase alfa was designed to improve fat absorption and nutritional status in patients whose endogenous lipase production is insufficient. Despite showing efficacy in Phase 2 trials for fat absorption in cystic fibrosis patients, the development program was discontinued in 2014 after a Phase 3 trial (the SURE study) in preterm infants failed to meet its primary endpoint.

Brand names

Kiobrina

Other names

recombinant human bile salt-stimulated lipasebile salt-activated lipaseBSSL