GC Biopharma’s Hunterase ICV gets orphan drug designation

GC Biopharma said Thursday that the Ministry of Food and Drug Safety (MFDS) recently granted Hunterase ICV (intracerebroventricular) orphan drug designation for treating severe Hunter syndrome.

The company previously received orphan drug designation for Hunterase ICV from Russia in 2017, Japan in 2020, and Europe in 2021.

(GC Biopharma’s headquarters office in Yongin, Gyeonggi Province

Hunterase ICV is the only treatment worldwide that improves central nervous system symptoms by delivering medication directly into the ventricles of the brain using a device inserted into the head. This delivery method allows the drug to reach the patient's blood vessels and central nervous system cells, relieving symptoms caused by central nervous system damage, including cognitive loss and delayed physical and motor development. About 70 percent of patients with Hunter syndrome worldwide have severe central nerve damage.

GC Biopharma was the first in the world to obtain a license for Hunterase ICV, a treatment for severe Hunter syndrome administered intraventricularly, in Japan in 2021 and the second in Russia in November 2024. Hunterase ICV is also in phase 1 clinical trials in Korea.

“As Hunterase ICV is designated as an orphan drug in Korea, we will strive to address the unmet needs of patients with severe Hunter syndrome,” said Lee Jae-woo, head of the Development Headquarters GC Biopharma.

Hunter syndrome is a rare congenital disease that causes skeletal abnormalities and low intelligence due to a deficiency of the IDS (Iduronate-2-sulfatase) enzyme and is said to occur at a rate of one in 100,000 to 150,000 boys.