GC Green Cross Submits Clinical Phase 1 Trial Plan for Rare Disease Treatment to FDA

GC Green Cross announced on the 22nd that it has applied to the U.S. Food and Drug Administration (FDA) for an Investigational New Drug (IND) application and Fast Track designation for GC1130A, a treatment for Sanfilippo Syndrome Type A (MPS IIIA) co-developed with Nobelpharma.

Exterior view of GC Green Cross headquarters in Yongin-si, Gyeonggi Province
[Photo by GC Green Cross]

The Fast Track program is designed to accelerate drug development for serious diseases with unmet medical needs. Being designated as a Fast Track drug provides the benefit of regular communication with the FDA.

GC Green Cross plans to accelerate the development of GC1130A through this IND and Fast Track application. Global clinical trials will be conducted in the United States, leading the effort, as well as in South Korea and Japan. Phase 1 clinical trials will evaluate the safety and tolerability of GC1130A.

Sanfilippo Syndrome Type A is an autosomal recessive genetic disorder caused by a gene defect that leads to the accumulation of heparan sulfate in the body, causing progressive damage. Severe brain damage is a major symptom, and most patients die around the age of 15. It is a severe rare disease with no approved treatments, resulting in significant unmet medical needs for patients.

GC Green Cross, in collaboration with Nobelpharma, is developing GC1130A as an enzyme replacement therapy (ERT) that directly administers heparan N-sulfatase?an enzyme not expressed in the patient’s body?into the brain ventricles (intracerebroventricular injection, ICV) to treat brain lesions in patients with Sanfilippo Syndrome Type A. Based on efficacy and safety demonstrated in preclinical stages, GC1130A has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. FDA, and recently also received ODD from the European Medicines Agency (EMA).

A representative from GC Green Cross stated, "We will accelerate new drug development through rapid clinical entry via collaboration between the two companies to bring hope to patients suffering from Sanfilippo Syndrome Type A worldwide.”