Vega Therapeutics Receives FDA Orphan Drug Designation for VGA039 for the Treatment of von Willebrand Disease

Vega Therapeutics, a clinical-stage biotechnology company developing novel therapies for rare blood disorders, today announced that the US Food and Drug Administration (FDA) has granted the company orphan drug designation for VGA039 for the treatment of the rare bleeding disorder, von Willebrand disease (VWD).

VGA039 is a first-in-class antibody therapy with a novel mechanism of action that modulates Protein S – a key co-factor involved in thrombin generation during both the initiation and propagation phases of coagulation. By promoting thrombin generation through targeting Protein S, VGA039 addresses a fundamental mechanism of clot formation in VWD and, as a subcutaneously self-administered antibody therapy, has potential to transform VWD treatment.

“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.”

An ongoing Phase 1 clinical study (NCT05776069) is designed to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic profile of VGA039 in healthy volunteers and VWD patients across sites in the EU and US. In preclinical studies, VGA039 has demonstrated efficacy in VWD, as well as in numerous congenital bleeding disorders.