Kedrion's Plasma-Derived Treatment Receives Orphan Drug Designation for Rare Genetic Disorder
In a significant advancement for patients with rare genetic disorders, the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) to Kedrion's experimental treatment for congenital Aceruloplasminemia (ACP). This condition is an autosomal recessive disorder caused by mutations in the ceruloplasmin (CP) gene, which leads to a deficiency or absence of ceruloplasmin. This plasma protein is crucial for iron transport in the body.
Without intervention, iron accumulates in key organs like the brain, liver, and pancreas, leading to severe complications such as neurological symptoms, diabetes, anemia, and retinal degeneration. The recognition from the EMA underscores Kedrion's commitment to addressing the unmet medical needs for rare and ultra-rare diseases, paving the way for potential new therapeutic options.
Kedrion, as a scientifically driven innovator, is pioneering ways to derive therapies from underutilized by-products of plasma fractionation. By converting industrial plasma waste into viable treatment avenues, the organization not only maximizes resource utilization but also champions sustainability in biopharmaceutical innovation.
Andrea Caricasole, head of Research, Development, and Innovation at Kedrion, shared insights into this milestone: "This EMA classification, following our recent Orphan Drug designation by the FDA, is a testament to our scientifically grounded approach to innovating in the sphere of rare and ultra-rare diseases. We are unlocking the therapeutic potential of ceruloplasmin to combat Aceruloplasminemia, a serious, extremely rare neurodegenerative disorder with no existing effective treatment options. For patients suffering from this rare condition, for whom no approved medication is available, this milestone represents a tangible step closer to the future availability of a new therapy. Our work illustrates how advanced research and sustainable practices can converge to develop impactful solutions."
This achievement is a result of collaborative efforts with partners from academia and the clinical field, and it is supported by Italy's Ministry of Enterprises and Made in Italy (MIMIT) through the NATURAL research project, which aims to drive research for new therapies utilizing squandered plasma fractionation intermediates.
The foundation for this breakthrough is robust, as it builds on two significant scientific publications that reflect Kedrion's vision. A study published in Nature Communications Biology (2024) demonstrated the feasibility of purifying ceruloplasmin from unused plasma fractionation intermediates and highlighted its therapeutic efficacy in preclinical models of Aceruloplasminemia. Additionally, the Lancet eBioMedicine (2025) provided functional insights into the missense variants of the ceruloplasmin gene and assessed the actual prevalence of Aceruloplasminemia using population data.
Together, these studies underscore the clinical potential and the urgent need for targeted therapies for this extremely rare condition.
Kedrion's commitment to the clinical development of its treatment for Aceruloplasminemia in Europe continues, aiming to afford timely access to patients currently lacking effective therapeutic options.
Kedrion Biopharma specializes in collecting and fractionating blood plasma to manufacture and distribute plasma-derived therapies for rare, ultra-rare, and serious conditions, including coagulation and neurological disorders, immunodeficiencies, and Rh sensitization. Employing approximately 5,200 individuals worldwide, the company operates a network of plasma collection centers in the United States and the Czech Republic, as well as production sites in five countries. Kedrion is dedicated to creating a world where science and supply know no boundaries, collaborating with the medical-scientific community, institutions, patient advocacy groups, and research establishments to foster innovation and improve care. Every connection we make impacts someone, somewhere.
Without intervention, iron accumulates in key organs like the brain, liver, and pancreas, leading to severe complications such as neurological symptoms, diabetes, anemia, and retinal degeneration. The recognition from the EMA underscores Kedrion's commitment to addressing the unmet medical needs for rare and ultra-rare diseases, paving the way for potential new therapeutic options.
Kedrion, as a scientifically driven innovator, is pioneering ways to derive therapies from underutilized by-products of plasma fractionation. By converting industrial plasma waste into viable treatment avenues, the organization not only maximizes resource utilization but also champions sustainability in biopharmaceutical innovation.
Andrea Caricasole, head of Research, Development, and Innovation at Kedrion, shared insights into this milestone: "This EMA classification, following our recent Orphan Drug designation by the FDA, is a testament to our scientifically grounded approach to innovating in the sphere of rare and ultra-rare diseases. We are unlocking the therapeutic potential of ceruloplasmin to combat Aceruloplasminemia, a serious, extremely rare neurodegenerative disorder with no existing effective treatment options. For patients suffering from this rare condition, for whom no approved medication is available, this milestone represents a tangible step closer to the future availability of a new therapy. Our work illustrates how advanced research and sustainable practices can converge to develop impactful solutions."
This achievement is a result of collaborative efforts with partners from academia and the clinical field, and it is supported by Italy's Ministry of Enterprises and Made in Italy (MIMIT) through the NATURAL research project, which aims to drive research for new therapies utilizing squandered plasma fractionation intermediates.
The foundation for this breakthrough is robust, as it builds on two significant scientific publications that reflect Kedrion's vision. A study published in Nature Communications Biology (2024) demonstrated the feasibility of purifying ceruloplasmin from unused plasma fractionation intermediates and highlighted its therapeutic efficacy in preclinical models of Aceruloplasminemia. Additionally, the Lancet eBioMedicine (2025) provided functional insights into the missense variants of the ceruloplasmin gene and assessed the actual prevalence of Aceruloplasminemia using population data.
Together, these studies underscore the clinical potential and the urgent need for targeted therapies for this extremely rare condition.
Kedrion's commitment to the clinical development of its treatment for Aceruloplasminemia in Europe continues, aiming to afford timely access to patients currently lacking effective therapeutic options.
About Kedrion Biopharma
Kedrion Biopharma specializes in collecting and fractionating blood plasma to manufacture and distribute plasma-derived therapies for rare, ultra-rare, and serious conditions, including coagulation and neurological disorders, immunodeficiencies, and Rh sensitization. Employing approximately 5,200 individuals worldwide, the company operates a network of plasma collection centers in the United States and the Czech Republic, as well as production sites in five countries. Kedrion is dedicated to creating a world where science and supply know no boundaries, collaborating with the medical-scientific community, institutions, patient advocacy groups, and research establishments to foster innovation and improve care. Every connection we make impacts someone, somewhere.
Topics Health）
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