3 November 2021 - - The European Medicines Agency has granted orphan drug designation to Hunterase ICV (intracerebroventricular, generic name: idursulfase-beta (recombinant)) for the treatment of mucopolysaccharidosis type II (Hunter syndrome) in the European Union, South Korea-based GC Pharma (006280.KS) said.

Hunterase ICV, developed by GC Pharma as the world's first Hunter syndrome therapy by ICV injection, is delivered directly to the cerebral Parenchyma, allowing it to reach the cells of the brain and central nervous system.

GC Pharma has received manufacturing and marketing approval for Hunterase ICV in Japan on January 2021.

The decision by the EMA to grant orphan designation to Hunterase ICV comes from the Committee for Orphan Medicinal Products' positive opinion.

The COMP at the EMA has issued a positive opinion based on data from the phase I/II study of Hunterase ICV.

The data demonstrated significant decreasing (≥ 70%) of heparan sulfate in cerebrospinal fluid, regarded as the key factor for delayed cognitive development while also showing positive effects on improving the developmental age.

In addition, Hunterase ICV meet the all criteria to obtain this designation including medical plausibility.

The COMP recognized that Hunterase ICV could provide "significant benefits" to patients compared to existing IV-administered treatments approved in Europe.

Mucopolysaccharidosis type II (Hunter syndrome) is a genetic lysosome disease that occurs mainly in boys.

It is a systemic disease causing a variety of symptoms of the nerves, bones, joints and organs due to a congenital deficiency in lysosomes, which are enzymes with the role of breaking down unnecessary lipids and sugars in the body.

It has been reported that in serious cases, patients often become bedridden in their mid-teens. The incidence in Japan is one in every 100,000-150,000 people, and there is no definitive cure.

The current standard treatment is enzyme replacement therapy, together with symptomatic therapy.

GC Pharma (formerly known as Green Cross Corp.) is a biopharmaceutical company that offers protein therapeutics and vaccines.

Headquartered in Yongin, South Korea, GC Pharma is a plasma protein and vaccine product manufacturer globally.

Green Cross Corp. updated its corporate brand to GC Pharma in early 2018. Green Cross Corp. remains the company's legal name.