{"id":8976,"date":"2025-11-27T10:27:20","date_gmt":"2025-11-27T09:27:20","guid":{"rendered":"https:\/\/www.kedrion.it\/?post_type=news&p=8976"},"modified":"2025-11-27T10:37:16","modified_gmt":"2025-11-27T09:37:16","slug":"ema-grants-orphan-drug-designation-to-our-plasma-derived-treatment-for-aceruloplasminemia-a-rare-genetic-disorder-of-iron-metabolism","status":"publish","type":"news","link":"https:\/\/www.kedrion.it\/en\/ema-grants-orphan-drug-designation-to-our-plasma-derived-treatment-for-aceruloplasminemia-a-rare-genetic-disorder-of-iron-metabolism\/","title":{"rendered":"EMA grants Orphan Drug Designation to our plasma-derived treatment for Aceruloplasminemia, a rare genetic disorder of iron metabolism"},"content":{"rendered":"\n\n\n\n\n

European Medicines Agency (EMA)<\/strong> has granted Orphan Drug Designation (ODD)<\/strong> to our investigational treatment <\/strong>for Congenital Aceruloplasminemia (ACP)<\/strong>, a rare genetic disorder of iron metabolism.<\/p>\n\n\n\n

Aceruloplasminemia <\/strong>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<\/strong>: Orphanet https:\/\/www.orpha.net\/en\/disease\/detail\/48818<\/a><\/p>\n\n\n\n

EMA\u2019s decision highlights our role as a science-driven innovator<\/strong> 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<\/strong>. This approach not only optimizes the use of a precious resource but also advances sustainability<\/strong> in biopharmaceutical innovation.<\/p>\n\n\n\n

Andrea Caricasole<\/strong>, Chief R&D and Innovation Officer at Kedrion, said:
\u201cThis designation from EMA, following the recent Orphan Drug Designation granted by the FDA, is a further validation of our science-driven approach<\/strong> to rare and ultra-rare disease innovation. By unlocking the therapeutic potential of this protein, we aim to address Congenital 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, <\/strong>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.\u201d<\/em><\/p>\n\n\n\n

This milestone is the result of collaborative efforts with academic and clinical partners<\/strong> and is supported by the Italian Ministry for Enterprises and Made in Italy (MIMIT)<\/strong> through the research project named \u201cNATURAL\u201d<\/strong>, which aim to foster research for new therapies by leveraging unused plasma fractionation intermediates.<\/p>\n\n\n\n

This achievement builds on two recent high-impact scientific publications that reflect the vision behind Kedrion\u2019s approach. Nature Communications Biology (2024)<\/strong> 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)<\/strong> 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. <\/p>\n\n\n\n

Kedrion will continue to advance this treatment for Aceruloplasminemia toward clinical development<\/strong> in Europe, with the goal of enabling timely access for patients who currently have no effective treatment options.<\/p>\n","protected":false},"featured_media":8977,"template":"","categories_news":[],"class_list":["post-8976","news","type-news","status-publish","has-post-thumbnail","hentry"],"acf":[],"yoast_head":"\nEMA grants Orphan Drug Designation to our plasma-derived treatment for Aceruloplasminemia, a rare genetic disorder of iron metabolism - Kedrion Biopharma<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.kedrion.it\/en\/ema-grants-orphan-drug-designation-to-our-plasma-derived-treatment-for-aceruloplasminemia-a-rare-genetic-disorder-of-iron-metabolism\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"EMA grants Orphan Drug Designation to our plasma-derived treatment for Aceruloplasminemia, a rare genetic disorder of iron metabolism - 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