TargED Biopharmaceuticals’ TGD001 receives Orphan Drug Designation in the United States of America
TargED Biopharmaceuticals B.V., a clinical-stage private biotechnology company focused on developing improved treatments for thrombotic diseases, today announces that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead product TGD001, a von Willebrand factor targeted thrombolytic, for the treatment of thrombotic thrombocytopenic purpura (TTP). This designation follows the received TGD001’s Orphan Drug status in the European Union (granted in July 2024), further solidifying TGD001’s promise to address the unmet medical need for patients worldwide. TGD001 is currently tested in a Phase 1 study to evaluate TGD001’s safety, tolerability and pharmacokinetics in healthy volunteers (EU CT ID number: 2024-514931- 63-00). First safety read-out from the Phase 1 study is expected by mid-2025.
“We are excited to receive Orphan Drug Designation for TGD001 from the FDA now as well” said Kristof Vercruysse, CEO of TargED Biopharmaceuticals. “. With the added support of both the FDA and EMA, TargED Biopharmaceuticals is ready to accelerate clinical trials in both regions, aiming to bring our innovative therapy to treat TTP patients as quickly as possible.”
Orphan Designation is granted by FDA to drug products intended for the treatment, prevention, or diagnosis of rare diseases that affect fewer than 200,000 people in the United States. Benefits of an orphan designation include a 7-year market exclusivity upon marketing authorization, regulatory fee reductions, and potential accelerated review and fast-track pathways for bringing the drug to the market more quickly.
About TTP
TTP is a rare, life-threatening blood disorder characterized by sudden episodes of clot-formation in small blood vessels throughout the body. This leads to disruption of the blood flow to organs such as the kidney, heart, and brain, resulting in the functional failure of these organs. Patients experiencing a TTP attack present with fatigue, breathing difficulties, bleeding underneath the skin or bleeding in general, and, in severe cases, neurological dysfunction leading to a seizure, stroke, or coma. Between 11,370 and 22,803 people are currently affected by TTP in the USA. Despite recent advances, 15% of iTTP patients who receive treatment do not survive, and half of those who live still suffer long term health impairment.
About TGD001
TGD001 is being developed to offer patients with clotting disorders a faster, safer and more effective treatment than today’s standard of care – saving lives and reducing disability. TGD001 is a groundbreaking thrombolytic designed to break down clots of all sizes and compositions – from large clots which cause AIS, down to ‘micro-clots’ which cause conditions such as the rare, life-threatening disorder TTP. TGD001 is a first-in-class ‘fusion protein’ drug with a unique two-step mode of action. It targets clots via an antibody fragment that binds to a non-functional domain of von Willebrand Factor (VWF), a protein present in all types of thrombi. Then, in the immediate vicinity of the thrombus, it activates the endogenous enzyme system that degrades both VWF and fibrin, two key clotting ingredients which form the structural bond in the thrombus. This mode of action enables rapid and effective thrombolysis without systemic activation of the lysis cascade and the resulting bleeding risk. TGD001 is administered as short or bolus injection, making it a suitable therapeutic for the emergency, in-patient and first-response setting.
We would like to express our gratitude to the researchers, healthcare professionals, and our amazing team who have contributed to the development of TGD001. The company remains steadfast in its mission to transform the lives of patients with rare and severe diseases through cutting-edge science and innovation.
About TargED Biopharmaceuticals
TargED Biopharmaceuticals B.V. is a Netherlands based biotechnology company that develops first-inclass biological drugs to improve treatment of thrombosis. TargED stands for Targeted Enzyme Delivery. Their biological drugs are unique by using antibodies fragments (“VhH”) to deliver enzymes to sites of thrombosis, enabling ‘targeted’ thrombolysis. Its lead compound, TGD001, is currently tested in a first in human clinical trial and will be further developed for the treatment of thrombotic thrombocytopenic purpura (TTP) and Acute Ischemic Stroke (AIS). The objective is to accelerate thrombolysis in all forms of thrombosis, irrespective of the thrombus composition. TargED, a spin-off of the University Medical Center Utrecht, was founded in July 2020. Since then, TargED raised more than €45 million of funding, supporting its growth to become a clinical-stage company. For more information, please visit www.targedbio.com
Source: TargED news
