Vega Therapeutics, Inc., a clinical-stage biotechnology company, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to VGA039 for the treatment of von Willebrand disease (VWD). This designation marks a significant step in the development of a novel therapy for this rare bleeding disorder.
VGA039 is a first-in-class antibody therapy that modulates Protein S, a key cofactor involved in thrombin generation during both the initiation and propagation phases of coagulation. By targeting Protein S, VGA039 aims to promote thrombin generation, addressing a fundamental mechanism of clot formation in VWD. The therapy is designed for subcutaneous self-administration, offering the potential to transform VWD treatment by reducing the burden of frequent intravenous infusions associated with current therapies.
Novel Mechanism of Action
VGA039's mechanism of action involves attenuating Protein S cofactor function for tissue factor pathway inhibitor α (TFPIα) and activated protein C (aPC). This augmentation and restoration of thrombin generation during both the initiation and propagation phases of coagulation addresses fundamental deficiencies of clot formation in VWD.
Clinical Development
An ongoing Phase 1 clinical study (NCT05776069) is evaluating the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of VGA039 in healthy volunteers and VWD patients across sites in the EU and US. Preclinical studies have demonstrated VGA039's efficacy in VWD, as well as in numerous congenital bleeding disorders.
Von Willebrand Disease: An Unmet Need
Von Willebrand disease (VWD) is a bleeding disorder characterized by defective or low amounts of von Willebrand factor (VWF), leading to insufficient platelet adhesion and unstable clot formation. VWD can cause severe bleeding, potentially damaging organs and significantly impacting patients’ daily lives. Current treatments are limited and often involve factor replacement therapies requiring frequent intravenous (IV) infusions.
Expert Commentary
"Receiving orphan drug designation status is an important step for VGA039," said Gary Patou, MD, Chief Medical Officer of Vega Therapeutics. "VGA039 has the potential to be a new treatment for VWD with a profile to reduce the treatment burden for people living with this disease."
