GC Pharma applies for US fast track designation for treatment of rare brain disease caused by death at age 15

by times news cr

2024-04-24 02:01:21

Joint development with Nobel Pharma of a new drug to treat Sanfilippo Syndrome Type A
Application for phase 1 clinical trial (IND)/fast track designation to the U.S. FDA
Promoting development of enzyme replacement therapy treatment administered directly into the ventricle
Unmet medical demand due to lack of cure ↑

GC Green Cross announced on the 23rd that it has applied to the U.S. Food and Drug Administration (FDA) for a phase 1 clinical trial protocol (IND) and fast track designation for ‘GC1130A’, a type A treatment for Sanfilippo syndrome that is being jointly developed with Nobel Pharma.

Fast Track is a system operated by the FDA to accelerate the development of treatments for serious diseases with large unmet medical needs. Once designated as a fast-track product, development time can be shortened by allowing regular communication with the FDA. When applying for product approval, a reduction in review time can also be expected. In addition, the fast track designation alone receives market and industry attention as it recognizes the potential for drug demand.

GC Green Cross expects that this application will speed up the development of GC1130A. Clinical trials are planned to be conducted in the United States, Korea, and Japan. It is said that the phase 1 clinical trial was designed with a focus on evaluating safety and tolerability.

Sanfilippo syndrome (type A) is a recessive genetic disease in which heparan sulfate accumulates in the body due to a genetic defect, causing gradual damage. It is a serious rare disease in which severe brain damage is the main symptom and most patients die around the age of 15. It is also a disease with a huge unmet medical need as there is no approved treatment yet.

GC Green Cross is developing a new drug jointly with Nobel Pharma, a domestic bio company specializing in orphan drugs. We are currently developing Enzyme Replacement Therapy (ERT), which involves intracerebroventricular injection (ICV) of enzymes that are not expressed in the patient’s body. By proving its efficacy and safety in the non-clinical stage, it was designated as an orphan drug (ODD) and a rare pediatric drug (RPDD) by the FDA, and was recently designated as an ODD by the European Medicines Agency (EMA).

A GC Pharma official said, “We will do our best to speed up the development of new drugs by quickly entering clinical trials so that we can provide hope to patients around the world suffering from Sanfilippo Syndrome Type A.”

Kim Min-beom, Donga.com reporter [email protected]

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2024-04-24 02:01:21

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