#Italy #Orphan Drug #Castelvecchio Pascoli #Kedrion #Aceruloplasminemia

Kedrion's Milestone in Rare Disease Treatment

Kedrion Biopharma has achieved a significant milestone in the realm of rare diseases as the European Medicines Agency (EMA) has officially granted Orphan Drug Designation (ODD) to their investigational plasma-derived treatment for congenital aceruloplasminemia. This rare genetic disorder affects iron metabolism, and the ODD highlights a crucial step forward towards addressing unmet medical needs in rare and ultra-rare diseases.

Understanding Aceruloplasminemia

Aceruloplasminemia is an autosomal recessive disorder caused by mutations in the ceruloplasmin gene (CP), leading to a deficiency or complete absence of ceruloplasmin—a vital plasma protein necessary for iron transport. Without appropriate treatment, excessive iron accumulates in the brain, liver, and pancreas, which can result in severe neurological symptoms, diabetes, anemia, and retinal degeneration.

The implications of this condition are profound, as patients experience a debilitating decline in health due to the lack of effective treatment options. Thus, Kedrion’s progress in developing a solution is not merely a company success but a beacon of hope for those affected.

Kedrion's Innovative Approach

The significance of the EMA's designation lies in Kedrion's innovative approach to treatment development. The company leads the way in creating therapies from unused plasma fractionation intermediates, transforming industrial plasma waste into potential new therapies. This approach optimizes the use of valuable resources while promoting sustainability within biopharmaceutical innovation.

Andrea Caricasole, the head of R&D and Innovation at Kedrion, stated: "The EMA designation, following the recent Orphan Drug Designation granted by the FDA, reinforces our scientific approach to innovation in rare and ultra-rare diseases. By harnessing the therapeutic potential of this protein, we aim to address aceruloplasminemia, an ultra-rare and devastating neurodegenerative disorder that currently lacks effective options. For patients with this orphan disease who currently have no approved drug, this milestone represents a tangible advancement towards future therapy availability. Our efforts demonstrate how advanced research and sustainable practices can converge to offer relevant solutions."

Kedrion’s achievement results from collaborative efforts with academic and clinical partners, supported by the Italian Ministry of Enterprise and Made in Italy (MIMIT) through the 'NATURAL' research project. This initiative focuses on fostering research into new therapies by utilizing previously unused plasma fractionation intermediates.

Scientific Foundations

Underpinning this progress are important scientific publications that reflect Kedrion’s vision. A study published in Nature Communications Biology in 2024 demonstrated the feasibility of purifying ceruloplasmin from unused plasma fractionation intermediates and its therapeutic efficacy in preclinical models of aceruloplasminemia. Additionally, a study in Lancet eBioMedicine in 2025 provided insights into functional aspects of nonsense variants of the ceruloplasmin gene and assessed the real-world prevalence of aceruloplasminemia using population data.

Together, these studies underscore the clinical potential, strong momentum towards scientific understanding, and the urgency of developing targeted therapies for this ultra-rare disease.

Next Steps for Kedrion

Kedrion is committed to driving the development of this aceruloplasminemia treatment through clinical phases in Europe, aiming to ensure timely access for patients currently lacking effective therapeutic options.

About Kedrion Biopharma

Kedrion Biopharma specializes in collecting and fractionating blood plasma to produce and distribute plasma therapies for rare, ultra-rare, and debilitating diseases, including coagulation and neurological disorders, immune deficiencies, and Rh sensitization. The company employs approximately 5,200 people globally, with plasma collection centers in the U.S. and the Czech Republic and production facilities across five countries. Kedrion is dedicated to creating a world where science and healthcare know no bounds by collaborating with the medical-scientific community, institutions, patient advocacy groups, and research organizations to drive innovation and improve healthcare. Every connection we make impacts someone, somewhere.