GC Biopharma expands Asian footprint with Hunter Syndrome approvals in India, Taiwan

SOUTH KOREA—GC Biopharma, a South Korean pharmaceutical company, has secured marketing authorization for its Hunter syndrome treatment, Hunterase IV, in India and Taiwan, expanding access to the therapy across Asia.

The company said India’s Central Drugs Standard Control Organisation (CDSCO) approved the intravenous (IV) formulation, while Taiwan’s Food and Drug Administration (TFDA) approved both Hunterase IV and Hunterase ICV.

In Taiwan, Hunterase ICV is marketed under the brand name Irifaze ICV and is administered through intracerebroventricular infusion.

Expanding access across Asia

Following the latest regulatory approvals, Hunterase IV is now authorized in 14 countries, while Hunterase ICV has approvals in four countries.

GC Biopharma has already established a presence in major Asian markets, including Japan, China, and Malaysia, and the new approvals strengthen its position in the region’s rare disease market.

India represents a potentially significant market for Hunterase IV because relatively few patients with Hunter syndrome currently receive available treatments.

The approval therefore gives GC Biopharma an opportunity to address an unmet need among patients who have limited access to enzyme replacement therapy.

Meanwhile, Taiwan’s approval of both Hunterase IV and Hunterase ICV provides access to two treatment approaches.

The company said Taiwan has established systems for early screening of Hunter syndrome and patient support, creating a relatively accessible environment for rare disease treatment.

“These approvals are significant as they address the unmet medical needs of local Asian patients who previously lacked sufficient treatment options,” said Eun-Chul Huh, CEO of GC Biopharma.

“We will continue to dedicate our efforts to improving the quality of life for patients worldwide.”

Hunterase ICV targets central nervous system complications

Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), is a rare inherited disorder caused by a deficiency of lysosomal enzymes needed to break down glycosaminoglycans (GAGs).

As these substances accumulate in cells and tissues, patients can develop skeletal abnormalities, cardiovascular complications, and progressive cognitive impairment.

The disorder predominantly affects males and occurs in approximately one in 100,000 to 150,000 live male births.

Hunterase IV is administered intravenously as an enzyme replacement therapy.

However, the therapeutic enzyme has limited ability to cross the blood-brain barrier (BBB), restricting its effect on the central nervous system (CNS).

In contrast, Hunterase ICV delivers the enzyme directly into the cerebral ventricles, bypassing the BBB and reaching the CNS.

GC Biopharma developed the approach to address neurological manifestations associated with Hunter syndrome, including cognitive decline.

The company estimates that about 70% of patients with Hunter syndrome have severe disease accompanied by CNS impairment.

With the latest regulatory decisions, GC Biopharma plans to continue expanding access to its Hunterase portfolio in international rare disease markets.

Sign up HERE to receive our email newsletters with the latest news and insights from Africa and around the world, and follow us on LinkedIn for updates.