GC Biopharma has received approval for the first treatment in South Korea for Hunter syndrome patients with central nervous system symptoms. This innovative therapy directly administers medication to brain tissue, overcoming limitations of existing intravenous treatments. It’s expected to broaden treatment options for patients with this rare disease.
On Friday, GC Biopharma announced that its brain-infusion Hunter syndrome treatment, Hunterraze ICV, has been approved by the Ministry of Food and Drug Safety. This marks a milestone as the first approved treatment in South Korea targeting Hunter syndrome patients with central nervous system symptoms.
Hunter syndrome is a rare genetic disorder caused by an enzyme deficiency, resulting in sugar accumulation in the body. Symptoms include skeletal abnormalities, organ dysfunction, and cognitive decline. Some patients experience central nervous system damage, leading to cognitive impairment and behavioral issues.
While existing intravenous (IV) treatments effectively address systemic symptoms, they struggle to reach brain lesions due to the blood-brain barrier (BBB).
Hunterraze ICV tackles this challenge by delivering medication directly into the brain’s ventricles. It’s designed to manage both systemic and central nervous system symptoms when used alongside existing IV formulations.
Clinical trials have shown the drug’s effectiveness in reducing heparan sulfate, a substance linked to central nervous system damage. A five-year follow-up study in Japan revealed sustained low levels of this marker and observed delays or improvements in cognitive decline. Extended clinical trials in South Korea are ongoing, with a minimum four-year follow-up period.
Hunterraze ICV is the world’s first brain-infusion treatment for Hunter syndrome. It has already gained approval in Japan, Russia, Peru, and Taiwan, with GC Biopharma pursuing expansion into additional markets.
GC Biopharma Chief Executive Officer (CEO) Heo Eun-cheol emphasized that this approval is a significant breakthrough, offering a new treatment option for patients with neuropathic Hunter syndrome in South Korea. It remains committed to addressing unmet medical needs for patients with rare diseases.
In related news, GC Biopharma has inked a technology export deal worth up to 634.7 billion KRW (about 475 million USD) for LA-GLA, a next-generation Fabry disease treatment candidate co-developed with Hanmi Pharmaceutical.
GC Biopharma and Hanmi Pharmaceutical have entered an exclusive licensing agreement with global rare disease specialist BioMarin Pharmaceutical for LA-GLA.
The deal includes an upfront payment of 70 million USD and potential milestone payments of up to 405 million USD during development and commercialization stages. Additional royalties are expected after the product launch.