GC Biopharma (006280.KS) is expanding into the Latin American market with its rare disease treatment "Hunterase ICV." Following approvals in Japan and Russia, the company has now secured regulatory approval in Peru, accelerating its global market expansion.
GC Biopharma announced Thursday that its intracerebroventricular (ICV) Hunter syndrome treatment "Hunterase ICV" has received product approval from Peru's General Directorate of Medicines, Supplies and Drugs (DIGEMID).
The approval marks the third overseas authorization for the drug, following Japan and Russia. The company plans to continue pursuing entry into additional countries, including other Latin American markets.
Hunter syndrome is a congenital rare disease in which glycosaminoglycans accumulate in the body due to a deficiency of the IDS enzyme. The disease causes skeletal abnormalities, joint deformities, respiratory and cardiac dysfunction, and cognitive decline. It primarily affects boys, occurring in approximately one in 100,000 to 150,000 births.
About two-thirds of patients exhibit a severe form accompanied by central nervous system damage. As the disease progresses, patients experience cognitive decline and behavioral abnormalities, significantly affecting their quality of life and prognosis.
Hunterase ICV is administered directly into the brain's ventricles once a month. It was developed to address the limitations of conventional intravenous enzyme replacement therapy, which cannot cross the blood-brain barrier and is therefore limited in improving central nervous system symptoms.
In Japanese clinical trials, the treatment significantly reduced heparan sulfate, a key substance responsible for central nervous system damage. Patients' cognitive and developmental functions tended to remain stable or improve, and the effects were sustained in long-term follow-up observations, the company said.
With this approval, GC Biopharma plans to expand its global presence based on the clinical data accumulated in the rare disease field. The company is expected to focus on addressing unmet needs among patients with severe neurological Hunter syndrome, for whom treatment options remain limited.
"Based on the long-term clinical data we have secured, we will continue to address the unmet medical needs of patients with severe Hunter syndrome," said Lee Jae-woo, head of development at GC Biopharma.
