GC said Tuesday that the European Medicines Agency (EMA) has granted orphan drug designation (ODD) to Hunterase ICV (intracerebroventricular), its treatment of severe hunter syndrome using a ventricular injection.

The EMA based its decision on the clinical data of Hunterase ICV, which showed that it could give “significant benefit” to patients compared to the existing IV therapy approved in Europe, according to the company.

Hunter syndrome (mucopolysaccharidosis type II) is an inherited lysosomal storage disease that occurs primarily in boys. It causes an enzyme deficiency that interferes with the body's ability to break down certain complex sugars, resulting in severe skeletal, tissue, neurological, and multi-organ complications and, ultimately, death. It is known to occur in one out of 150,000 people, with about 70 percent of patients showing symptoms of central nerve damage.

In a clinical trial conducted in Japan, Hunterase ICV reduced heparan sulfate, a key causative agent of central nerve damage, by more than 70 percent and demonstrated a positive effect on improving the age when the illness can develop.

Hunterase ICV also met all other criteria for ODD, such as the disease's prevalence rate of fewer than five patients per 10,000 people and medical plausibility.

"This designation once again proved that Hunterase ICV can solve the unmet needs of patients with Hunter syndrome," GC CEO Huh Eun-chul said. "We will continue our efforts to make meaningful changes in the lives of people with Hunter syndrome around the world."

Hunterase ICV is a treatment that inserts a device into a patient's head and directly administers the drug to the ventricle. GC stressed that the method is revolutionary as drugs in the existing intravenous formulation do not pass through the blood-brain barrier (BBB) and cannot reach the cerebral parenchyma.