South Korean biopharmaceutical company GC Pharma (KRX:006280) disclosed on Wednesday that it plans to commercialise Hunterase (Idursulfase-beta) ICV for the treatment of Hunter syndrome in Japan under an exclusive licensing agreement with Clinigen KK, a wholly owned subsidiary of Clinigen Group plc (LON:CLIN).

Hunter syndrome (Mucopolysaccharidosis type II) is an inherited lysosomal storage disease that occurs primarily in boys. It causes an enzyme deficiency that interferes with the body's ability to break down certain complex sugars, resulting in serious skeletal, tissue, neurological and multi-organ complications and ultimately, death.

GC Pharma's Hunterase (Idursulfase-beta) ICV is a human recombinant iduronate-2-sulfatase (IDS) used in enzyme replacement therapy. The intravenous injection does not penetrate the blood-brain barrier in clinically adequate amounts, but GC Pharma's product is delivered directly to cerebral ventricles by intracerebroventricular (ICV) administration, in order to reach the cells of the brain and central nervous system.

The company said that Hunterase (Idursulfase-beta) ICV is expected to meet the unmet needs of severe patients in improving their quality of life, as a method that can achieve what previous intravenous injection could not.

A Phase 1/2 clinical trial conducted as an investigator-initiated trial by Prof. Torayuki Okuyama in the National Center for Child Health and Development in Japan showed a significant decrease in Heparan sulfate which causes mental retardation.