GC Pharmaceuticals has secured regulatory approvals in India and Taiwan for its Hunter syndrome treatment, Hunterase IV, alongside an approval for its direct-to-brain intracerebroventricular formulation, Hunterase ICV, from Taiwan’s health authorities. According to an announcement by the company on August 24, 2026, the approvals expand the global reach of Hunterase IV to 14 countries while bringing Hunterase ICV to four international markets.
Global Regulatory Approvals Expand Access for Hunter Syndrome Patients
The Central Drugs Standard Control Organization (CDSCO) in India and the Taiwan Food and Drug Administration (TFDA) both granted marketing authorization for Hunterase IV, known generically as idursulfase-beta. Simultaneously, the Taiwan TFDA cleared Hunterase ICV for use. Prior to these decisions, GC Pharmaceuticals had secured approvals for the intravenous formulation in key markets including Japan, China, and Malaysia.
Jaewoo Lee, head of the development division at GC Pharmaceuticals, stated according to company communications that the approvals address significant unmet medical needs for Asian patients who previously faced a shortage of treatment options. Lee emphasized that the company remains committed to enhancing the quality of life for those diagnosed with the condition.
Understanding Hunter Syndrome and Treatment Mechanisms
Hunter syndrome, or mucopolysaccharidosis type II, is a rare, inherited genetic disorder caused by a deficiency in the lysosomal enzyme responsible for breaking down glycosaminoglycans (GAG). The accumulation of GAGs leads to progressive cellular damage, resulting in physical manifestations such as skeletal abnormalities, cardiac dysfunction, and cognitive decline. The condition primarily affects males, occurring at an estimated rate of 1 in every 10만~15만 명 중 1명 live births.
To combat this neurological impact, Hunterase ICV utilizes a direct intracerebroventricular administration method designed to deliver the therapeutic enzyme straight into the central nervous system. Medical data indicates that approximately 70% of all Hunter syndrome patients suffer from severe forms of the disease characterized by central nervous system deterioration.
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