GC Green Cross, Rare Bleeding Disorder Treatment Designated as FDA Orphan Drug

GC Green Cross announced on the 5th that its internally developed candidate drug for Thrombotic Thrombocytopenic Purpura (TTP), GC1126A, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA).

The ODD designation date for GC1126A was on the 27th of last month, local time. Typically, when designated as an orphan drug, benefits such as tax reductions on research and development costs, exemption from approval review fees, and exemption from submitting an Initial Pediatric Study Plan can be received. Additionally, upon approval, a 7-year market exclusivity period from the date of marketing authorization is granted.

TTP is a rare bleeding disorder occurring in approximately 3 to 11 individuals per one million people. Small blood clots form throughout the body, blocking blood flow to major organs such as the brain and heart, and if not properly treated, about 90% of patients die. The pathogenesis is known to be caused by a deficiency or functional impairment of the protein-degrading enzyme 'ADAMTS13' due to autoantibodies.

GC1126A is a mutated protein that evades autoantibodies against ADAMTS13 while increasing its half-life. At the ISTH 2023 held in June, it was announced that GC1126A demonstrated superior efficacy and maintained higher activity compared to existing drugs and wild-type ADAMTS13.

A GC Green Cross official stated, "We are focusing on securing data with the goal of developing the best treatment within the rare bleeding disorder category," adding, "We will do our best to develop innovative new drugs to provide patients with new treatment options."

This content was produced with the assistance of AI translation services.