GC Biopharma said Tuesday that the U.S. Food and Drug Administration (FDA) has approved its investigative new drug (IND) application for phase 1 clinical trial of “GC1130A” to treat Sanfilippo syndrome or mucopolysaccharidosis type IIIA (MPS IIIA), which is being developed in collaboration with Novel Pharma.

With this approval, the companies will begin a multinational clinical trial in the U.S., Korea, and Japan later this year to evaluate the safety and tolerability of GC1130A in a global clinical trial, GC Biopharma added.

GC Biopharma is developing an enzyme replacement therapy (ERT) for treating brain lesions in MPS IIIA patients by administering an enzyme (heparan N-sulfatase) not expressed in the patient's body directly into the ventricles of the brain (intracerebroventricular injection or ICV).

The treatment has been granted orphan drug designation (ODD) and rare pediatric disease designation (RPDD) by the U.S. Food and Drug Administration and ODD by the European Medicines Agency based on demonstrated efficacy and safety in nonclinical studies.

Sanfilippo syndrome (type A) is a recessive genetic disorder caused by a gene defect that leads to a buildup of heparan sulfate in the body, which causes progressive damage. It is a severe and rare disease with severe brain damage as the main symptom, and most patients die around the age of 15. There are no licensed treatments for the disease, and the unmet medical needs of patients are high.

"With this approval, we can quickly enter phase 1, and we will do our best to develop the new drug to give hope to patients suffering from the disease," a GC Biopharma official said.