Announcing Marketing Approval for Hunterase ICV Injection 15 mg, the World’s First Enzyme Replacement Therapy for Mucopolysaccharidosis Type II (Hunter Syndrome) Administered by ICV Injection

YONGIN, South Korea--(BUSINESS WIRE)--GC Pharma (formerly known as Green Cross Corporation) (KRX: 006280), a South Korean biopharmaceutical company, and Clinigen K.K. (“Clinigen”), headquartered in Tokyo, has received on January 22, 2021, Japan manufacturing and marketing approval for Hunterase ICV (intracerebroventricular) Injection 15 mg (generic name: idursulfase-beta (recombinant)) as a treatment for mucopolysaccharidosis type II (Hunter syndrome).

An enzyme-replacement therapy drug for intravenous injection is already in use in Japan and other countries as a treatment for systemic symptoms of mucopolysaccharidosis type II. However, there is no therapeutical drug targeting the central nervous system symptoms, from which approximately 70% of patients suffer.

The newly approved Hunterase ICV Injection 15 mg, developed by GC Pharma, is delivered directly to the cerebral ventricles, allowing it to reach the cells of the brain and central nervous system. It is expected to alleviate aspects of the disease such as delayed psychomotor development.

In the result of the phase I/II study conducted by Dr. Torayuki Okuyama, principal investigator of the study, Department Head of Clinical Laboratory Medicine of the National Center for Child Health Development, Hunterase demonstrated a significant decrease of heparan sulfate in cerebrospinal fluid, which is regarded as the key factor for delayed cognitive development.

The approval in Japan is ahead of the world, being the first approval of Hunterase ICV Injection in any country worldwide. Hunterase ICV Injection 15 mg is also the world’s first and only drug for the treatment of central nervous system symptoms of mucopolysaccharidosis type II.

Eun Chul Huh, Ph.D., President, GC Pharma, said:

“This approval represents a great triumph of community involvement to address the significant unmet need of slowing the cognitive decline in MPS II patients. It is a result of patients, physicians and other collaborative efforts.”

Takashi Matsuki, Representative Director and General Manager, Clinigen K.K., said:

“We are delighted to have received manufacturing and marketing approval for this groundbreaking new drug, through our partnership with GC Pharma, in keeping with our group’s mission of ‘THE RIGHT MEDICINE. THE RIGHT PATIENT. THE RIGHT TIME’. We are striving to provide Hunterase to patients with mucopolysaccharidosis type II in Japan as soon as possible.”

About mucopolysaccharidosis type II

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 50,000 people, and there is no definitive cure. The current standard treatment is enzyme replacement therapy (ERT), together with symptomatic therapy.

About Clinigen K.K.

Clinigen K.K. is a fully owned subsidiary of Clinigen plc (listed on London’s Alternative Investment Market), a company engaged in a range of pharmaceutical-related business, with its headquarters in Burton-on-Trent, UK. In Japan, Clinigen K.K. works on the development, manufacture and marketing of orphan drugs and pharmaceuticals for niche markets.

In collaboration with our fellow group company, Link Healthcare, headquartered in Chuo-ku, Tokyo, Clinigen K.K. provides an early-access program (EAP) for Japanese pharmaceutical manufacturers to supply unapproved drugs for humanitarian use, together with consulting services to facilitate the use of this program.

About GC Pharma

GC Pharma is a biopharmaceutical company that delivers life-saving and life-sustaining protein therapeutics and vaccines. Headquartered in South Korea, GC Pharma is one of the largest protein products manufacturers in the world and has been dedicated to quality healthcare solutions more than half a century. Green Cross Corporation updates its corporate brand as GC Pharma in early 2018. Green Cross Corporation remains the company's registered, legal name.

This release includes forward-looking statements, which express the current beliefs and expectations of GC Pharma’s management. Such statements speak only as of the date on which they are made and the company undertakes no obligation to update or revise any forward-looking statement, whether as a result of new information, future events or otherwise.