GC Biopharma Applies For US FDA Fast Track Designation For Sanfilippo Syndrome Treatment
GC Biopharma Applies For US FDA Fast Track Designation For Sanfilippo Syndrome Treatment
  • BK Min
  • 승인 2024.04.24 19:35
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GC Biopharma, led by CEO Eun-cheol Heo, has applied for Fast Track Designation from the U.S. Food and Drug Administration (FDA) for 'GC1130A', a treatment for Sanfilippo Syndrome Type A (MPS IIIA). This treatment is being developed in collaboration with Novel Pharma. The company has submitted a phase 1 clinical trial protocol (IND) and fast-track designation application to the FDA and awaits approval.

Sanfilippo Syndrome is a rare and severe genetic disease. Patients with this condition experience gradual damage, due to an accumulation of heparan sulfate in the body caused by a genetic defect. Severe brain damage is the main symptom, and most patients die around the age of 15. Currently, there is no approved treatment for this disease.

Fast Track is a system created by the FDA to speed up drug development for serious diseases with large unmet medical needs. If 'GC1130A' is designated as a Fast Track, the company will benefit from regular communication with the FDA. This IND and Fast Track application is expected to speed up clinical trials and new drug development for ‘GC1130A’, a treatment for MPS IIIA.

GC Biopharma is developing an enzyme replacement therapy (ERT) for MPS IIIA patients, in collaboration with Novel Pharma. The treatment uses intracerebroventricular injection (ICV) of an enzyme (heparan N sulfatase) that is not expressed in the patient's body to treat brain lesions in MPS IIIA patients. The company plans to conduct global clinical trials in Korea and Japan, starting with the United States, to evaluate the safety and tolerability of ‘GC1130A’ through phase 1 clinical trials.

The treatment received Orphan Drug (ODD) and Orphan Drug (RPDD) designation from the U.S. FDA based on efficacy and safety proven in the non-clinical stage. It also recently received an Orphan Drug (ODD) designation from the European EMA.

GC Biopharma official said, "We will accelerate the development of new drugs by quickly entering clinical trials through collaboration between the two companies to provide hope to patients worldwide suffering from Sanfilippo Syndrome Type A"

The link to the Korean version of this article is as below.

▷GC녹십자, 산필리포 증후군 치료제 美 FDA 패스트트랙 지정 신청

Source_ⓒGC Biopharma


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