GC Biopharma said that the intracerebroventricular (ICV) enzyme replacement therapy (ERT) drug for Sanfilippo Syndrome Type A, jointly developed with Korean biotech venture Novel Pharma, has been designated as an orphan drug by the European Medicines Agency (EMA).

The recognition follows the drug's previous designations by the U.S. Food and Drug Administration (FDA) as both a rare pediatric disease drug and an orphan drug last year.

Sanfilippo Syndrome Type A is a severe, recessive genetic disorder characterized by the accumulation of heparan sulfate in the central nervous system, leading to progressive neurological damage.

Most patients with this condition tragically pass away around the age of 15, and currently, there are no approved treatments available, leaving a significant unmet medical need.

Since 2020, GC Biopharma and Novel Pharma have been collaboratively developing an ERT, which involves the direct administration of the enzyme heparan N-sulfatase (not naturally produced in patients) into the brain's ventricles (ICV).

Their research has led to the successful production of a highly concentrated enzyme formulation suitable for ICV administration. The companies have also secured numerous patents related to this therapy, both domestically and internationally.

The drug's production is based on GC Biopharma's proprietary recombinant protein manufacturing technology. Both companies plan to jointly proceed with clinical trials in the near future.

"Following recognition in the U.S., this designation in Europe further acknowledges our pipeline's potential to address the unmet need of the disease,” a GC official said. “We are committed to expediting clinical entry to bring this treatment to patients as quickly as possible."

The orphan drug designation in Europe brings several benefits, including reduced fees for marketing authorization applications, tax reductions, and, upon market authorization, an exclusivity period of ten years, which is extended to twelve for pediatric treatments.