GC Biopharma's Sanfilippo treatment eligible for FDA priority review
Jung-Eun Kim
Jan 18, 2023 (Gmt+09:00)
GC Biopharma headquarters GC Biopharma Corp. said on Wednesday that its Sanfilippo type A treatment, which is under joint development with Novel Pharma, a bio-venture specializing in orphan drugs, has received rare pediatric disease designation (RPDD) from the US Food and Drug Administration.
This disease is a genetic defect that causes progressive damage due to the accumulation of heparan sulfate in the central nervous system. It is a severe disease with most patients dying at the age of 15 as there is no approved treatment yet.
The two companies have developed enzyme replacement therapy (ERT)-based treatments since 2020, in which enzymes are injected into patients with enzyme deficiency.
According to the company's explanation, once it receives the RPDD designation from the FDA, it can apply for a "priority review voucher," which is the eligibility to shorten the new drug approval review period to six months.
"Based on the know-how we have gained in the field of orphan drugs, we will continue our efforts to provide better treatment options to patients suffering from rare diseases," a GC Biopharma official said.