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GC Pharma Announces EMA Grants Orphan Drug Designation to Hunterase ICV, The World’s First Enzyme Replacement Therapy for Mucopolysaccharidosis Type II Administered by ICV Injection

YONGIN, South Korea--(BUSINESS WIRE)--GC Pharma (006280.KS) today announced that the European Medicines Agency (EMA) granted orphan drug designation (ODD) to Hunterase ICV (intracerebroventricular, generic name: idursulfase-beta (recombinant)) for the treatment of mucopolysaccharidosis type II (Hunter syndrome) in the European Union.

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’ (COMP) 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.

“This designation will be an important milestone toward addressing the urgent, unmet needs of families and patients living with Hunter syndrome,” said EC Huh, Ph.D, President of GC Pharma. “We will continue to make meaningful changes in the lives of patients with rare diseases around the world.”

About Hunter syndrome

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 1 in every 100,000-150,000 people, and there is no definitive cure. The current standard treatment is enzyme replacement therapy (ERT), together with symptomatic therapy.

About GC Pharma

GC Pharma (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers life-saving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Pharma is one of the leading plasma protein and vaccine product manufacturers globally and has been dedicated to quality healthcare solutions for more than half a century. Green Cross Corporation updated its corporate brand to GC Pharma in early 2018. Green Cross Corporation remains the company's legal name.

This press release may contain forward-looking statements, which express the current beliefs and expectations of GC Pharma's management. Such statements do not represent any guarantee by GC Pharma or its management of future performance and involve known and unknown risks, uncertainties and other factors. GC Pharma undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule.


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