GC Biopharma and Novel Pharma Receive FDA IND Approval for MPS IIIA Treatment 'GC1130A'

21 May 2024 | Tuesday | News

Global Clinical Trials Set to Begin This Year, Aiming to Prove Safety and Tolerability of Groundbreaking Sanfilippo Syndrome Therapy
Image Source : Public Domain

Image Source : Public Domain

GC Biopharma 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."


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