GC Biopharma Corp. announced on Oct. 5 that their therapeutic candidate for Thrombotic Thrombocytopenic Purpura (TTP), “GC1126A,” has received a Rare Drug Designation from the U.S. Food and Drug Administration (FDA).
As a result of this designation, GC1126A will benefit from tax deductions on research and development costs, exemptions from licensing review fees, and an exemption from submitting pediatric study plans. Furthermore, once approved, it will enjoy market exclusivity rights for seven years from the date of authorization.
TTP is a rare bleeding disorder that occurs in approximately 3-11 out of every million individuals. The disorder involves the formation of small blood clots throughout the body, which can block blood flow to major organs such as the brain and heart. If not treated properly, about 90% of the patients can succumb to this lethal condition. It is known to be caused by a deficiency or functional reduction of the protein-cleaving enzyme ADAMTS13, either due to its absence or from auto-antibodies.
GC1126A is a mutated protein that not only evades any auto-antibodies against ADAMTS13 but also has an extended half-life. At the 2023 conference of the International Society on Thrombosis and Haemostasis (ISTH) in June it was announced that GC1126A maintains superior efficacy and high activity compared to existing drugs and wild-type ADAMTS13.