Clotilia®

Verenafusp alfa is a recombinant modified human iduronate-2-sulfatase lysosomal enzyme protein covalently linked to a Fab fragment of a monoclonal antibody to the human insulin receptor.

Verenafusp alfa is synthesized using recombinant DNA technology in a CHO cell line.

Clotilia® is used as an enzyme replacement therapy for the treatment of children and adults with Hunter syndrome (Mucopolysaccharidosis II) who have a deficiency or reduced level of the iduronate-2-sulfatase enzyme in the body.

Clotilia® possesses the properties of the human iduronate-2-sulfatase enzyme. Upon entering the affected cells of the body's organs and tissues, including the brain, it breaks down glycosaminoglycans (dermatan sulfate and heparan sulfate) and prevents their accumulation. The drug helps to stabilize the course of the disease and reduce the clinical manifestations of Hunter syndrome, including improvements in the condition of the central nervous system.