When designated as an orphan drug, there are incentives such as tax credits for clinical development costs and exemptions from PDUFA user fees Initial Pediatric Study Plan. Additionally, upon receiving marketing approval, a 7-year period of market exclusivity is granted.

TTP is a rare blood disorder that affects approximately 3 to 11 individuals per 1 million people. It is a life-threatening condition characterized by the formation of small blood clots throughout the body, obstructing blood flow to critical organs, including the brain and heart. If left untreated, TTP can result in a mortality rate as high as 90%. The development of TTP is associated with a deficiency of the proteolytic enzyme ADAMTS13 in the patient's body (congenital TTP) or the sudden production of antibodies that inhibit the enzyme activity (immune-mediated TTP).

GC1126A is a novel ADAMTS13 mutein designed to evade autoantibodies with an extended half-life. As presented at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress in June, non-clinical data demonstrated efficacy of the drug candidate in disease models while maintaining higher activity levels when compared to existing treatments or wild-type ADAMTS13.

Speaking on this achievement, GC Biopharma stated, "We are dedicated to collecting data to develop a Best-in-Class treatment for such rare disorders. We remain committed to our mission of providing patients with new treatment options by continuously developing innovative drugs."