GC Pharma’s Hunter syndrome treatment receives orphan drug designation in EU

GC Pharma announced Tuesday that the European Medicines Agency has granted orphan drug designation to its Hunterase ICV, a novel treatment for mucopolysaccharidosis type II, also known as the Hunter syndrome.

The designation brings a variety of significant advantages under the EMA’s effort to encourage the development of drugs to treat rare diseases or conditions.

GC Pharma said the company will be able to speed up regulatory procedures to market Hunterase ICV in Europe.

Hunterase ICV can be administered directly into the brain of a patient. By inserting a device in the head, the drug can reach the brain cells and central nervous system, according to the company.

With existing intravenous formulations, the drugs were unable to penetrate the blood-brain barrier and thus unable to reach the cerebral parenchyma, the functional tissue in the brain.

GC Pharma CEO Huh Eun-chul said the company will continue to make efforts to address the urgent, unmet needs of families and patients living with Hunter syndrome.

In January, GC Pharma received manufacturing and marketing approval for Hunterase ICV in Japan.

By Shim Woo-hyun (ws@heraldcorp.com)