GC Green Cross, Severe Hunter Syndrome Treatment Designated as European Orphan Drug

[Asia Economy Reporter Seo So-jeong] GC Green Cross (CEO Heo Eun-cheol) announced on the 2nd that its severe Hunter syndrome treatment 'Hunterase ICV (intracerebroventricular)', administered via intracerebroventricular injection, has been designated as an 'orphan drug' by the European Medicines Agency (EMA).

Hunter syndrome is a congenital rare disease caused by a deficiency of the enzyme 'IDS (Iduronate-2-sulfatase)', leading to skeletal abnormalities and intellectual disability. It occurs in approximately 1 in 100,000 to 150,000 male births. Generally, about 70% of patients are known to exhibit central nervous system damage symptoms.

Hunterase ICV improves upon the existing intravenous (IV) treatment method, which cannot cross the patient's blood-brain barrier (BBB) and thus fails to reach the brain parenchymal tissue. GC Green Cross was the first in the world to successfully develop the ICV administration treatment method and obtained product approval in Japan earlier this year.

The company stated that in this approval, Hunterase ICV was recognized to provide 'significant benefits' to patients compared to the existing IV treatment approved in Europe, based on clinical data. In clinical trials conducted in Japan, Hunterase ICV reduced heparan sulfate, a key causative substance of central nervous system damage, by more than 70% and showed positive effects on developmental age improvement.

Additionally, Hunterase ICV met all criteria for European orphan drug approval, including 'prevalence (5 or fewer per 10,000 people)' and 'medical plausibility.'

Heo Eun-cheol, CEO of GC Green Cross, said, "Through this designation, Hunterase ICV has once again proven that it can address the unmet needs of Hunter syndrome patients," adding, "We will continue our efforts to create meaningful changes in the lives of Hunter syndrome patients worldwide."

This content was produced with the assistance of AI translation services.