Vega Therapeutics Receives FDA Orphan Drug Designation for VGA039 for the Treatment of von Willebrand Disease
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).
- 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).
- 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.
- In preclinical studies, VGA039 has demonstrated efficacy in VWD, as well as in numerous congenital bleeding disorders.