GC Biopharma said on the 6th that it has applied to Peru's drug regulator for approval of its Hunter syndrome treatment "Hunterase ICV." Hunter syndrome is a rare disease in which enzyme deficiency causes skeletal abnormalities and cognitive decline. Patients experience deformities such as a protruding forehead and die before age 30. The name comes from the Canadian physician Hunter who first identified it.
Hunterase ICV involves implanting a device in the head to deliver the drug directly into the brain's ventricles. Seventy percent of Hunter syndrome patients also have central nervous system damage. Hunterase ICV delivers the drug to the patient's ventricles and boosts treatment efficacy. It addresses the issue of conventional injection formulations potentially failing to cross the blood-brain barrier. According to clinical data from Japan, Hunterase ICV reduced heparan sulfate, which causes central nervous system damage. It also improved patients' cognitive function or slowed deterioration.
Hunterase ICV is being sold commercially in Japan and Russia. In Aug. last year, the company applied to the Ministery of Food and Drug Safety for approval. The company said it plans to expand approvals to Southeast Asia, the Middle East, and Central and South America.
Earlier, in 2012, GC Biopharma succeeded in developing a treatment for Hunter syndrome. Before the world's second successful development of a treatment, prices neared several million won per injection. That monopoly ended with the launch of GC Biopharma's Hunterase. It is credited with improving treatment conditions for patients.
GC Biopharma aims to develop second and third versions of Hunterase. For rare diseases, once the first new drug is successfully developed, diverse orphan drug support policies by country allow for rapid commercialization. Lee Jae-u, head of development at GC Biopharma, said, "We have presented a new treatment alternative for patients with severe Hunter syndrome," adding, "We will continue research and development in the field of rare diseases."