The European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to Kedrion’s investigational therapy for the treatment of Congenital Factor V Deficiency, a rare inherited bleeding disorder.
The condition is characterized by a deficiency of Factor V, an essential protein in blood the coagulation cascade. Patients experience a wide spectrum of bleeding symptoms that can vary from mild to severe. With limited therapeutic options available, patients today rely mainly on non-specific treatments. (Source: Orphanet)
This designation represents an important step in advancing the development of Kedrion’s Factor V program.
Kedrion’s research leverages its expertise in plasma-derived therapies, including the valorization of unused or under-used plasma fractionation intermediates to unlock new treatment possibilities.
Andrea Caricasole, Chief Research & Innovation Officer, commented: “This milestone confirms the importance of our work in rare hematology and our ongoing commitment to developing innovative therapies for patients with unmet medical needs.”