GC Biopharma Corp. (006280.KS) has announced that the European Medicines Agency (EMA) granted Orphan Drug Designation (ODD) to GC1130A, an intracerebroventricular (ICV) Enzyme Replacement Therapy (ERT) candidate developed in collaboration with Novel Pharma for Sanfilippo Syndrome type A (mucopolysaccharidosis type IIIA).

Previously, GC1130A achieved significant milestones by securing Rare Pediatric Disease designation (RPDD) and Orphan Drug Designation (ODD) from the U.S. FDA in January 2023. The recent approval by the EMA marks another substantial accomplishment for the therapy in Europe.

Sanfilippo Syndrome (type A) is a genetic disorder causing central nervous system damage due to the accumulation of Heparan sulfate, leading to progressive neurodegeneration in the pediatric population. Patients with MPS IIIA typically have a life expectancy of around 15 years, and currently, there are no approved therapies available. In nonclinical studies, GC1130A has demonstrated both safety and efficacy in eliminating Heparan sulfate and restoring brain function.

Since 2020, GC Biopharma and Novel Pharma have collaborated on the development of GC1130A. Utilizing proprietary recombinant protein manufacturing technology, GC Biopharma has successfully formulated GC1130A to be compatible with cerebrospinal fluid at high protein concentrations. Novel Pharma, a biotech company specializing in rare disease drug development in Korea, has played a crucial role in this collaboration.

"This EMA designation further underscores the potential of our collaborative pipeline in addressing the disease pathology in upcoming clinical trials," stated GC Biopharma, emphasizing its commitment to advancing into clinical trials rapidly, recognizing the urgent and unmet medical needs of Sanfilippo Syndrome patients.

 

Source: prnewswire.com

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