GC Biopharma/Novel Pharma's Sanfilippo Syndrome Treatment Obtains FDA IND Approval

2024-05-20

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突破性疗法临床申请快速通道临床1期上市批准

YONGIN, South Korea, May 20, 2024 /PRNewswire/ -- GC Biopharma (006280. KS) and Novel Pharma announced that its jointly developed MPS IIIA 'GC1130A' treatment has received FDA IND approval.

With this approval, development of 'GC1130A' is expected to accelerate with multinational clinical trials commencing this year. Clinical trial sites have been set up in the US, Korea and Japan, and focus of the trial will center around proving the drug's safety and tolerability.

GC Biopharma and Novel Pharma have been co-developing ICV ERT (intracerebral ventricular enzyme replacement therapy) for MPS IIIA to address the lack of Heparan N Sulfatase expression in the patient's body.

'GC1130A' also received ODD and RPDD designations from the FDA and EMA during the pre-clinical phase on the back of robust safety and efficacy profile.

Sanfilippo Syndrome (type A) is a genetic disorder that triggers central nervous system damage by accumulating Heparan sulfate, leading to a progressive damage. If left untreated, it may result in a life-threatening outcome around the age of 15. With this approval, MPS III type A could become the first and only treatment option for patients with such disease.

GC Biopharma's representative further emphasized, "With this IND approval, we can now enter phase 1 clinical and we will do our utmost in new drug development to provide hope to patients suffering from the disease."

About GC Biopharma

Corp.

GC Biopharma Corp. (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers life-saving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Biopharma Corp. is one of the leading plasma protein and vaccine product manufacturers globally and has been dedicated to quality healthcare solutions for more than half a century.

This press release may contain biopharmaceuticals in forward-looking statements, which express the current beliefs and expectations of GC Biopharma's management. Such statements do not represent any guarantee by GC Biopharma or its management of future performance and involve known and unknown risks, uncertainties and other factors. GC Biopharma undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule.

GC Biopharma Corp. Contacts (Media)

Sohee Kim

[email protected]

Yelin Jun

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Rachel Kim

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SOURCE GC Biopharma

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