Posted by Michael Wonder on 22 Apr 2026
Star Therapeutics receives FDA rare paediatric disease and breakthrough therapy designations for VGA039 in von Willebrand disease prophylaxis
21 April 2026 - VGA039 is a once monthly, subcutaneously self-administered investigational therapy for the treatment of bleeding disorders, initially being evaluated for von Willebrand disease.
Star Therapeutics today announced that the US FDA has granted rare paediatric disease designation and breakthrough therapy designation to Star’s lead program, VGA039, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with von Willebrand disease.
