GC Biopharma said Wednesday that its intracerebroventricular (ICV) treatment for Hunter syndrome, Hunterase ICV, has received marketing authorization from Peru’s General Directorate of Medicines, Supplies and Drugs (DIGEMID).
The approval marks the company’s third overseas regulatory authorization for the therapy, following approvals in Japan and Russia. The company added that it is continuing efforts to expand into additional countries.
About two-thirds of Hunter syndrome patients are known to develop a severe phenotype accompanied by central nervous system damage. As the condition progresses, patients may experience cognitive decline and behavioral abnormalities, which can significantly affect quality of life and prognosis.
GC Biopharma headquarters in Yongin, Gyeonggi Province (Courtesy of GC Biopharma)
Hunterase ICV is administered directly into the brain ventricles once a month. Clinical trials in Japan found that the therapy significantly reduced heparan sulfate, which is linked to central nervous system damage in Hunter syndrome patients. The studies also showed that patients maintained or improved cognitive and developmental functions, and long-term follow-up confirmed sustained improvement in these areas.
“We will continue addressing the unmet medical needs of patients with severe Hunter syndrome based on the long-term clinical data we have secured,” said Lee Jae-woo, head of development at GC Biopharma.
Hunter syndrome is a rare inherited disorder caused by a deficiency of the IDS (iduronate-2-sulfatase) enzyme, leading to the accumulation of glycosaminoglycans (GAGs) in the body. The disease can cause skeletal abnormalities, joint deformities, respiratory and cardiac dysfunction, and cognitive impairment. It primarily affects boys, with an estimated incidence of one in every 100,000 to 150,000 male births.
Kim Ji-hye
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