GC Green Cross Rare Disease Treatment Designated for FDA Fast Track

GC Green Cross announced on the 10th that GC1130A, a treatment for Mucopolysaccharidosis Type IIIA (MPS IIIA) being co-developed with domestic biotech company Nobelpharma, has been designated as a Fast Track by the U.S. Food and Drug Administration (FDA).

GC Green Cross Headquarters Exterior Photo by GC Green Cross

Following the FDA's approval of the clinical Phase 1 trial application (IND) last month, the recent Fast Track designation is expected to accelerate the new drug development process.

Mucopolysaccharidosis Type IIIA is a rare genetic disorder caused by a gene defect that leads to the accumulation of heparan sulfate in the body, causing progressive damage. Most patients suffer severe brain damage and die around the age of 15.

GC1130A is a biopharmaceutical developed using Green Cross's high-concentration protein formulation technology administered to the central nervous system. Notably, it employs the intracerebroventricular (ICV) method, which involves direct administration into the brain ventricles to enhance therapeutic effects. The ICV method was first applied globally in Hunterase, Green Cross's treatment for Hunter syndrome.

GC1130A was previously designated as an orphan drug and pediatric orphan drug by the FDA last year, and this year it was also designated as a rare disease drug by the European Medicines Agency (EMA). Green Cross is preparing clinical trials in Korea, the United States, and Japan to evaluate the safety and tolerability of GC1130A.

A Green Cross representative said, “We are pleased that GC1130A has been designated as an FDA Fast Track drug in a situation where there is currently no approved treatment for Mucopolysaccharidosis. We will accelerate the development of this new drug to bring hope to patients suffering from Mucopolysaccharidosis.”

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