von Willebrand disease treatment gets FDA orphan drug designation

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blood bleeding disorders von Willebrand disease

The U.S. Food and Drug Administration (FDA) has granted Vega Therapeutics, Inc. orphan drug designation for its antibody therapy, VGA039, for the treatment of the rare bleeding disorder, von Willebrand disease (VWD).

Vega Therapeutics is a clinical stage biotechnology company developing novel therapies for rare blood disorders.

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. As a subcutaneously self-administered antibody therapy, it has the potential to transform VWD treatment, the company said.

“Receiving orphan drug designation status is an important step for VGA039,” said Gary Patou, 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 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 U.S. In preclinical studies, VGA039 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’ VGA039

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. 

Vega Therapeutics said VGA039 can be a universal hemostatic therapy to treat various bleeding disorders.

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