GC Biopharma Corp. has officially announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to its drug candidate, GC1126A, for the treatment of Thrombotic Thrombocytopenic Purpura (TTP). This significant development was finalized on September 27.

Receiving Orphan Drug Designation carries various incentives, including tax credits to offset clinical development expenses and exemptions from PDUFA user fees Initial Pediatric Study Plan. Furthermore, upon gaining marketing approval, a 7-year period of market exclusivity is conferred.

TTP is an uncommon blood disorder that impacts an estimated 3 to 11 individuals per 1 million in the population. It is a life-threatening condition characterized by the formation of small blood clots throughout the body, leading to the obstruction of blood flow to vital 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 linked to a deficiency of the proteolytic enzyme ADAMTS13 in the patient's body (congenital TTP) or the abrupt production of antibodies that inhibit the enzyme's activity (immune-mediated TTP).

GC1126A represents a groundbreaking ADAMTS13 mutein engineered to evade autoantibodies, featuring an extended half-life. Presented at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress in June, non-clinical data showcased the drug candidate's efficacy in disease models while maintaining higher activity levels compared to existing treatments or wild-type ADAMTS13.

In response to this achievement, GC Biopharma expressed their dedication to gathering data for the development of a Best-in-Class treatment for rare disorders. Their unwavering commitment remains focused on their mission to offer new treatment options to patients through the continuous development of innovative drugs.