Proteon Therapeutics, Inc., a privately held biopharmaceutical company developing novel, first-in-class pharmaceuticals to address the critical medical needs of patients with kidney and vascular diseases, today announced that its lead product, PRT-201, has received orphan drug designation in the European Union (EU) for the prevention of arteriovenous access dysfunction in hemodialysis patients. Proteon is investigating the use of PRT-201, a locally acting recombinant human elastase applied during surgical placement of a vascular access, to reduce vascular access failure. PRT-201 has previously received fast track and orphan drug designations from the U.S. Food and Drug Administration (FDA).
The European Medicines Agency grants orphan designation status to medicines intended to treat, prevent or diagnose diseases that are life-threatening or chronically debilitating in no more than five in 10,000 people in the EU. Based on its orphan status, Proteon is eligible to receive a 10-year market exclusivity period for PRT-201 in the EU, as well as other incentives, including protocol assistance and fee reductions.
"Vascular access is often considered the Achilles' heel of hemodialysis because of the high rates of failure and poor outcomes following surgical placement of an access," commented Timothy Noyes, president and chief executive officer of Proteon. "We believe that PRT-201 may provide significant clinical benefit to this orphan population, and we are therefore grateful for this recognition."
A functioning vascular access is a hemodialysis patient's lifeline, enabling the patient to undergo chronic hemodialysis. Unfortunately, arteriovenous fistulas (AVFs) and arteriovenous grafts (AVGs), the two forms of permanent vascular access, often experience patency loss, in which the access suffers from a significant reduction in or complete loss of blood flow, precluding hemodialysis. Patients can be subjected to further surgical or interventional procedures to restore blood flow, access abandonment and prolonged exposure to a dialysis catheter, which is associated with increased morbidity and mortality.