GC Biopharma announced on the 5th that the Ministry of Food and Drug Safety has designated the treatment for severe Hunter syndrome, 'Hunterase ICV (intracerebroventricular),' as an orphan drug.
Hunter syndrome is a congenital rare disease caused by a deficiency of the IDS (Iduronate-2-sulfatase) enzyme, leading to skeletal abnormalities and intellectual disability. It is reported to occur at a rate of 1 in every 100,000 to 150,000 boys.
Hunterase ICV is the only treatment globally that directly administers the drug to the brain ventricles using a device implanted in the head, aiming to improve central nervous system symptoms. The drug delivered in this manner alleviates symptoms related to central nervous system damage, such as cognitive decline and delayed physical development.
Approximately 70% of severe patients with Hunter syndrome exhibit central nervous system damage, and there has been a significant unmet medical demand as suitable treatments have not been developed.
In 2021, the company obtained the first-ever approval for 'Hunterase ICV,' a treatment for severe Hunter syndrome administered intraventricularly, in Japan. Following that, in November 2024, it received the second global approval in Russia. Hunterase ICV is currently undergoing Phase 1 clinical trials in South Korea. In 2017, it was designated as an orphan drug in Russia, followed by Japan in 2020, and Europe in 2021.
Lee Jae-woo, head of the development department at GC Biopharma, stated, "As Hunterase ICV has been designated as an orphan drug in South Korea, we will strive to address the unmet needs of patients with severe Hunter syndrome."