the von Willebrand disease drug gets FDA orphan designation


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

Vega Therapeutics is a clinical-stage biotechnology company developing new 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 cofactor involved in thrombin formation during the coagulation initiation and propagation phases. By promoting thrombin formation via targeting Protein S, VGA039 addresses the basic mechanism of blood clot formation in VWD. As a subcutaneous self-administered antibody therapy, it has the potential to transform the treatment of VWD, the company said.

“Accepting 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 burden of treatment for people living with this disease.”

An ongoing phase 1 clinical study designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic profile of VGA039 in healthy volunteers and VWD patients across sites in the EU and US. In preclinical studies, VGA039 demonstrated efficacy in VWD, as well as in many congenital bleeding disorders.

About von Willebrand’s disease

Von Willebrand’s disease (VWD) is a bleeding disorder in which the blood does not clot properly. In VWD, defective or low von Willebrand factor (VWF) leads to insufficient platelet adhesion and unstable clot formation. VWD causes severe bleeding that can damage organs and have a significant impact on the patient’s daily life. Current treatment is limited and includes factor replacement therapy which often requires intravenous (IV) infusions.

About VGA039 Vega Therapeutics

VGA039, is the first antibody therapy tailor-made for VWD with a novel mechanism of action targeting Protein S. By attenuating the function of Protein S cofactor for inhibitors of tissue factor α (TFPIα) and activated protein C (aPC) pathways, VGA039 augments and restores thrombin generation during coagulation initiation and propagation phases, overcoming the fundamental drawback of clot formation in VWD.

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


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