GC Pharma said on Wednesday that its Sanfilippo Syndrome Type A treatment, jointly being developed with bio-venture Novel Pharma, has been designated as a Rare Pediatrics Disease (RPD) by the U.S. FDA.

Sanfilippo syndrome (Type A), also known as mucopolysaccharidosis type IIIA (MPS IIIA), is a recessive genetic disease that causes gradual damage to the central nervous system (CNS) through the accumulation of heparin sulfate in the CNS due to genetic defects. It is a serious disease in which most patients die around the age of 15 due to worsening symptoms because there is no approved treatment yet.

The two companies have been jointly developing enzyme replacement therapy (ERT) for the disease since 2020. 

GC Pharma produces drugs at GMP facilities with its own recombinant protein production technology, and Novel Pharma conducts non-clinical studies. 

Both companies expect Orphan Drug Designation (ODD) to follow which should accelerate the development of the new drug through rapid clinical progress.

Under the current RPD scheme, the FDA awards priority review vouchers (PRV) to sponsors of rare pediatric disease product applications that meet certain criteria. Accordingly, if a new drug candidate is granted RPD designation, the PRV can help shorten the screening period to six months for future marketing applications of a different product.

Recently, the voucher has been valued at approximately $102 million.

"Based on the know-how we have accumulated in the field of rare medicines, we will continue to make efforts to provide better treatment options to patients suffering from rare diseases and their families," a GC Pharma official said.