Designated as an orphan drug in Korea following the US and Europe Affects 1 in 70,000 children… No approved treatment “Faster drug development expected to meet unmet medical needs”
GC Green Cross Headquarters
GC Green Cross announced on the 5th that its self-developed candidate for the treatment of Sanfilippo Syndrome Type A, 'GC1130A', has been designated as an orphan drug by the Ministry of Food and Drug Safety (MFDS) following the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
In South Korea, orphan drug designation is granted to medicines used for diseases affecting fewer than 20,000 people, medicines with no appropriate treatment alternatives, or medicines that significantly improve safety and efficacy compared to existing treatments. Once designated as an orphan drug domestically, benefits such as reduced pre-approval review fees and eligibility for conditional approval applications are provided.
Sanfilippo Syndrome is a type of Lysosomal Storage Disease known to occur at a rate of 1 in approximately 70,000 children. Typically, language development delays and developmental stagnation appear between ages 2 to 5, followed by cognitive decline, weakened motor skills, and respiratory issues, severely impacting life expectancy. Currently, there are no approved treatments for Sanfilippo Syndrome, and only rehabilitative therapies for symptom relief are available, indicating a high unmet medical need.
GC Green Cross is developing the treatment in an intracerebroventricular (ICV) formulation. By administering the drug directly into the ventricles, it is expected to be effective in improving cognitive function in severely affected patients. In fact, non-clinical results for GC1130A released last year demonstrated that the ICV formulation has up to 47 times higher drug delivery efficacy compared to direct intrathecal (IT) administration.
GC1130A is currently undergoing Phase 1 clinical trials in South Korea, the United States, and Japan. GC Green Cross aims to commercialize the treatment within five years.
Heo Eun-cheol, CEO of GC Green Cross, stated, “We expect the development speed to accelerate with the domestic orphan drug designation,” and added, “GC1130A will be a meaningful alternative to address the unmet medical needs of Sanfilippo Syndrome patients.”
Kim Min-beom
AI-translated with ChatGPT. Provided as is; original Korean text prevails.
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