GC Biopharma Corp. announced that its drug candidate, GC1126A, has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of Thrombotic Thrombocytopenic Purpura (TTP). This designation provides certain incentives to support the development of drugs for rare diseases:
- Tax Credits: Companies developing orphan drugs can receive tax credits for clinical development costs. These credits can help offset some of the expenses incurred during the drug development process.
- Exemption from PDUFA Fees: Orphan drug developers are exempt from paying certain user fees associated with the FDA’s Prescription Drug User Fee Act (PDUFA). This can result in cost savings for companies.
- Market Exclusivity: Upon receiving marketing approval, orphan drugs are granted a period of market exclusivity lasting seven years. During this time, other companies are generally unable to market a similar drug for the same indication, providing a competitive advantage.
TTP is a rare and life-threatening blood disorder characterized by the formation of small blood clots throughout the body, leading to the obstruction of blood flow to critical organs. The disorder can result from 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 and has an extended half-life. Non-clinical data have demonstrated its efficacy in disease models while maintaining higher activity levels compared to existing treatments or wild-type ADAMTS13.
Receiving Orphan Drug Designation is an important milestone for GC1126A, as it signifies recognition by the FDA of its potential to address the unmet medical needs of patients with TTP. The designation provides valuable support and incentives to continue its development and bring a promising treatment option to those affected by this rare and severe disorder.
About GC Biopharma Corp.
GC Biopharma Corp. is a biopharmaceutical company with a long history and a global presence. The company specializes in the development and manufacturing of plasma protein therapies and vaccines, contributing to the provision of quality healthcare solutions worldwide.
With a focus on delivering life-saving and life-sustaining products, GC Biopharma Corp. plays a crucial role in addressing medical needs and improving the health and well-being of individuals across the globe.