FDA Fast Tracks GCBP and GC1130A

YONGIN, South Korea, June 10, 2024 – GC Biopharma and Novel Pharma have announced that their jointly developed treatment for Sanfilippo syndrome Type A, designated GC1130A, has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA). This follows the recent FDA clearance of the investigational new drug (IND) application for GC1130A.

Understanding Sanfilippo Syndrome Type A

Sanfilippo syndrome Type A is a rare genetic disorder characterized by the harmful build-up of heparan sulfate in the central nervous system, leading to severe neurological degeneration. If left untreated, affected individuals often face critical health challenges by their mid-teens. GC1130A aims to be a pioneering treatment for this condition, offering hope where no specific therapy currently exists.

About GC1130A

GC1130A is an innovative biological drug developed using advanced high-concentration protein formulation technology. The drug is administered directly to the brain's ventricles through intracerebroventricular (ICV) injection. This method was first introduced globally by GC Biopharma with their Hunter syndrome treatment, Hunterase, which is approved in Japan.

Recognizing the significant medical needs of Sanfilippo syndrome patients, regulatory bodies have acknowledged the potential of GC1130A. In 2023, the FDA awarded the drug Rare Pediatric Disease Designation (RPDD) and Orphan Drug Disease (ODD) status. The European Medicines Agency (EMA) also granted it ODD status earlier this year.

Clinical Trials and Future Steps

GC Biopharma and Novel Pharma are gearing up to begin a multinational first-in-human clinical trial to assess the safety and tolerability of GC1130A. This trial will take place in Korea, the United States, and Japan.

FDA’s Fast Track Program

The FDA’s Fast Track program is designed to speed up the development and review of drugs aimed at treating serious conditions with unmet medical needs. This designation offers substantial support, including frequent interactions with the FDA throughout the stages of drug development and approval.

GC Biopharma and Novel Pharma expressed their satisfaction with the FDA’s Fast Track designation for GC1130A, underscoring the urgency of developing treatments for Sanfilippo syndrome. “This designation will enable us to accelerate the development of this new drug, bringing hope to patients and families affected by Sanfilippo syndrome,” the companies stated.

GC Biopharma: A Brief Overview

Formerly known as Green Cross Corporation, GC Biopharma is a biopharmaceutical firm specializing in lifesaving protein therapeutics and vaccines. Based in Yongin, South Korea, GC Biopharma is a leading global manufacturer of plasma protein and vaccine products, committed to high-quality healthcare solutions for over half a century.

About Novel Pharma

Novel Pharma is a burgeoning biotechnology company focused on creating first-in-class treatments for rare pediatric diseases using ICV administration. Headquartered in Seoul, South Korea, the company is actively developing therapies for MPSIIIA, GM1, MPSIVB, and Krabbe disease.

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!