The first enzyme replacement therapy for Hunter syndrome with ICV injections has been approved in Japan

Recently, Korean biopharmaceutical company GC Pharmaceuticals (formerly Green Cross Corporation) and Tokyo-based Cinigen K.K. announced that they had received Japanese approval on January 22, 2021 to manufacture and sell Hunterase Side Brain Indoor (ICV) injections 15 mg (common name: idursulfase-beta ) for the treatment of mucosal polysaccharine type II (Hunter syndrome).
type II is a hereditary lysosome disease that occurs mainly in boys.
It is a systemic disease that causes symptoms in nerves, bones, joints and organs due to a congenital lack of lysosomes, which are enzymes that break down unnecessary lipids and sugars in the body.
current standard treatments are enzyme replacement therapy (ERT) and symptom therapy.
February 2019, Sangamo Therapeutics announced the first "in vivo" human gene-editing therapy to permanently alter the DNA of patients with mucosal polysaccharide type II, and clinical trials using zinc finger nucleases for gene editing began at the same time.
two pharmaceutical companies, enzyme replacement drugs administered intravenously have been used in Japan and other countries to treat the systemic symptoms of type II. mucosal polysaccharide disease.
, however, there are currently no treatments for symptoms of the central nervous system, with about 70 per cent of patients suffering from symptoms of the central nervous system.
15mg of Hunterase ICV injection, approved by GC Pharmaceuticals, is delivered directly to the brain chamber, reaching brain cells and the central nervous system.
is expected to alleviate certain aspects of the disease, such as mental retardation.
Isursulfase beta ® a long-term treatment for patients with mucosal polysaccharid disease Type II.
idursulfase beta is a purified form of iduronate-2-sulfatase lysosome Malay acidase: recombinant human iduronate-2-sulfatase (rhI2S).
the effectiveness and safety of Hunterase was confirmed in two clinical studies that lasted 24-52 weeks, including male patients with Hunter syndrome between the ages of 38 months and 35 years.
The results of a Phase I/II study conducted by Dr. Torayuki Okuyama, lead researcher of the study and director of the Clinical Laboratory Of the National Center for The Healthy Development of Children, showed that Hunterase demonstrated a significant reduction in acetyl heparin sulfate in cerebrospinal fluid, which is considered a key factor in cognitive development.
this approval is the first time that any country in the world has approved Hunterase ICV injections.
15mg of Hunterase ICV injection is the world's first and only drug to treat symptoms of the central nervous system of mucosal polysaccharitis TYPE II.
source: Announcing Marketing Approval for Hunterase ICV Injection 15 mg, The World's First Enzyme Therapy for Mucopolysaccharidosis Type II (Hunter Syndrome) Directed by ICV Injection