Green Cross's 'Sanfilippo Syndrome Treatment' Designated as FDA Orphan Pediatric Drug

GC Green Cross Headquarters Exterior [Photo by GC Green Cross]

[Asia Economy Reporter Chunhee Lee] GC Green Cross announced on the 18th that the treatment for Sanfilippo Syndrome Type A (MPS III A), co-developed with the rare drug-specialized bio-venture Nobelpharma, has been designated as a Rare Pediatric Disease Drug (RPDD) by the U.S. Food and Drug Administration (FDA).

Sanfilippo Syndrome (Type A) is an autosomal recessive genetic disorder caused by a gene defect that leads to the accumulation of heparan sulfate in the central nervous system, causing progressive damage to the central nervous system. There is currently no approved treatment, and most patients suffer symptom worsening and die around the age of 15, making it a severe disease.

The two companies have been jointly developing an enzyme replacement therapy (ERT) for this disease since 2020. GC Green Cross produces the drug using its proprietary recombinant protein production technology at facilities compliant with Good Manufacturing Practice (GMP) standards. Nobelpharma is conducting non-clinical studies. Following the RPDD designation, an Orphan Drug Designation (ODD) is also anticipated, and the two companies plan to jointly accelerate clinical trials to speed up development.

With RPDD designation, when applying for approval later, companies can apply for a Priority Review Voucher (PRV), which can shorten the new drug approval review period to six months under special provisions. Typically, FDA new drug approval reviews take about 10 months. The PRV can also be used for approval applications of other substances besides the designated drug, and it can be sold or transferred. Recently, the U.S. rare disease gene therapy developer Bluebird Bio sold this voucher to the Dutch company Argenx for $102 million (approximately 126.5 billion KRW).

A GC Green Cross official said, “Based on the know-how accumulated in the rare drug field, we will continue efforts to provide better treatment options to patients and families suffering from rare diseases.”