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

May 23, 2023 |

2 min read

Vega Therapeutics , Inc., a clinical stage biotechnology company developing novel therapies for rare blood disorders, today announced that the U.S. 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).

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Vega Therapeutics, Inc., a clinical stage biotechnology company developing novel therapies for rare blood disorders, today announced that the U.S. 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.

About von Willebrand disease

Von Willebrand disease (VWD) is a bleeding disorder in which the blood does not clot properly. In VWD, defective or low amounts of von Willebrand factor (VWF) lead to insufficient platelet adhesion and unstable clot formation. VWD causes severe bleeding that can damage organs and lead to significant impact on patients’ daily lives. Current treatments are limited and include factor replacement therapies that require frequent intravenous (IV) infusions.

About Vega Therapeutics

Vega Therapeutics is a clinical stage biotechnology company developing novel, first-in-class therapies for rare blood disorders with overlooked patient needs, starting with von Willebrand disease (VWD). The company’s lead product candidate, VGA039, is the first purpose-built antibody therapy for VWD with a novel mechanism of action targeting Protein S. By attenuating Protein S cofactor function for tissue factor pathway inhibitor α (TFPIα) and activated protein C (aPC), VGA039 augments and restores thrombin generation during both the initiation and propagation phases of coagulation, addressing fundamental deficiencies of clot formation in VWD. VGA039 has potential to be a universal hemostatic therapy that can treat various bleeding disorders. For more information, please visit www.vega‑therapeutics.com and follow us on LinkedIn.