European Medicines Agency (EMA) grants Orphan Drug Designation to Kedrion's plasma-derived treatment for Congenital Aceruloplasminemia, a rare genetic disorder of iron metabolism
This designation marks a significant milestone for patients and reaffirms Kedrion's commitment to addressing unmet medical needs in rare and ultra-rare diseases
CASTELVECCHIO PASCOLI, Italy, Nov. 27, 2025 /PRNewswire/ -- Kedrion announces that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to its investigational treatment for Congenital Aceruloplasminemia (ACP), a rare genetic disorder of iron metabolism.
Aceruloplasminemia is an autosomal recessive disorder caused by mutations in the CP gene, leading to a deficiency or absence of Ceruloplasmin, a key plasma protein essential for iron transport. Without treatment, iron accumulates in the brain, liver, and pancreas, causing severe neurological symptoms, diabetes, anemia, and retinal degeneration. Source: Orphanet https://www.orpha.net/en/disease/detail/48818
EMA's decision highlights Kedrion's role as a science-driven innovator in the rare and ultra-rare disease space. By pioneering the development of therapies from unused plasma fractionation intermediates, Kedrion transforms industrial plasma waste into potential new treatments. This approach not only optimizes the use of a precious resource but also advances sustainability in biopharmaceutical innovation.
Andrea Caricasole, Chief R&D and Innovation Officer at Kedrion, said: "This designation from EMA, following the recent Orphan Drug Designation granted by the FDA, is a further validation of our science-driven approach to rare and ultra-rare disease innovation. By unlocking the therapeutic potential of this protein, we aim to address Aceruloplasminemia, a devastating ultra-rare and neurodegenerative condition that lacks effective options. For patients of this orphan disease, who currently do not have an approved drug, this milestone represents a concrete step forward in the future availability of a new therapy. Our work demonstrates how advanced research and sustainable practices can converge to deliver solutions that matter."
This milestone is the result of collaborative efforts with academic and clinical partners and is supported by the Italian Ministry for Enterprises and Made in Italy (MIMIT) through the research project named "NATURAL", which aim to foster research for new therapies by leveraging unused plasma fractionation intermediates.
This achievement builds on two recent high-impact scientific publications that reflect the vision behind Kedrion's approach. Nature Communications Biology (2024) demonstrated the feasibility of purifying Ceruloplasmin from unused plasma fractionation intermediates and its therapeutic efficacy in preclinical models of Aceruloplasminemia. Complementing this, Lancet eBioMedicine (2025) provided functional insights into missense variants of the Ceruloplasmin gene and assessed the real-world prevalence of Aceruloplasminemia using population-level data. Together, these studies reinforce clinical potential, a strong drive towards scientific understanding, and urgency of developing targeted therapies for this ultra-rare condition.
Kedrion will continue to advance this treatment for Aceruloplasminemia toward clinical development in Europe, with the goal of enabling timely access for patients who currently have no effective treatment options.
About Kedrion Biopharma
Kedrion Biopharma collects and fractionates blood plasma to produce and distribute plasma-derived therapies for rare, ultra-rare, and debilitating conditions like Coagulation and Neurological Disorders, Immunodeficiencies, and Rh sensitization. We employ approximately 5,200 people worldwide and our industrial network includes plasma collection centers in the United States and in the Czech Republic, and production facilities across 5 countries. We are committed to creating a world where science and care know no bounds, partnering with the medical-scientific community, institutions, patient advocacy groups, and research bodies to foster innovation and improve care. Every connection we make impacts someone, somewhere.
Logo: https://mma.prnewswire.com/media/2662888/Kedrion_Logo.jpg
Share this article