GC Biopharma's Sanfilippo treatment eligible for FDA priority review

The S.Korean company is entitled to request a shorter review period for the new drug's approval

GC Biopharma headquarters 
GC Biopharma headquarters 
Jung-Eun Kim 1
2023-01-18 17:12:53 likesmile@hankyung.com
Bio & Pharma

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.

Write to Jung-Eun Kim at likesmile@hankyung.com
#GC Biopharma #Green Cross Corporation #Novel Pharma #Sanfilippo type A treatment #rare pediatric disease designation #RPDD

GC Biopharma confirms safety of flu vaccine in phase 1 trial

GC Biopharma confirms safety of flu vaccine in phase 1 trial

GC Biopharma Hwasun plant South Korea's leading pharmaceutical company GC Biopharma Corp. said on Thursday that it has confirmed the safety and immunogenicity of the patch-type influenza vaccine MIMIX-Flu, which is under development jointly with Vaxess Technologies of the United States, in the

Green Cross' two Kosdaq-listed cell therapy arms to merge

Green Cross' two Kosdaq-listed cell therapy arms to merge

Green Cross's Hwasun factory in South Jeolla Province, South Korea South Korea's pharmaceutical group Green Cross Corp., or GC Pharma, will combine its two cell therapy developers listed on the junior Kosdaq market in efforts to scale up and sharpen its focus on the growing cell-based therapy m

Green Cross in talks with Russia to produce COVID-19 vaccine

Green Cross in talks with Russia to produce COVID-19 vaccine

Green Cross employees at Hwasun factory (Courtesy of Green Cross)  South Korea’s pharmaceutical company Green Cross Corporation (GC) is holding a series of close meetings on contract manufacturing of Russia’s COVID-19 vaccine CoviVac, according to industry sources on Mar. 17. C
(* comment hide *}