Vega Therapeutics, Inc.

Vega Therapeutics' VGA039 Receives FDA Orphan Drug Designation for Von Willebrand Disease

2 years ago

• Vega Therapeutics' VGA039, a first-in-class antibody, has been granted Orphan Drug Designation by the FDA for treating von Willebrand disease (VWD). • VGA039 modulates Protein S to enhance thrombin generation, addressing a core mechanism of clot formation in VWD. • A Phase 1 clinical study (NCT05776069) is underway in the EU and US to assess VGA039's safety, tolerability, and pharmacokinetics in healthy volunteers and VWD patients. • VGA039, designed for subcutaneous self-administration, aims to reduce the treatment burden associated with current VWD therapies.