GC Green Cross, Rare Disease 'Sanfilippo Syndrome' Treatment Phase 1 Clinical Trial Plan Approved in the US

GC Green Cross announced on the 20th that it has received approval from the U.S. Food and Drug Administration (FDA) for the clinical phase 1 trial plan (IND) of GC1130A, a treatment for Mucopolysaccharidosis type IIIA (MPS IIIA), which is being co-developed with the domestic bio-venture company Nobelpharma.

GC Green Cross Headquarters View [Photo by GC Green Cross]

Mucopolysaccharidosis type IIIA is a recessive genetic disorder caused by a gene defect that leads to the accumulation of heparan sulfate in the body, resulting in progressive damage. Severe brain damage is the main symptom, and most patients suffer from a severe rare disease that leads to death around the age of 15. There is currently no approved treatment, leaving a significant unmet medical need for patients.

With this IND approval, the development of GC1130A as a treatment for Mucopolysaccharidosis type IIIA is expected to accelerate. The two companies plan to start multinational clinical trials within this year in the United States, Korea, and Japan to evaluate the safety and tolerability of GC1130A.

A GC Green Cross representative stated, "With this approval enabling rapid entry into phase 1 clinical trials, we will do our best in drug development to bring hope to patients suffering from this disease.”