#Orphan Drug #Kedrion #Aceruloplasminemia

Kedrion's Groundbreaking Treatment Receives EMA Designation

In a significant development for patients suffering from a rare genetic disorder, the European Medicines Agency (EMA) has awarded Kedrion Biopharma the Orphan Drug Designation (ODD) for its investigational treatment targeting Congenital Aceruloplasminemia (ACP). This condition is characterized by a deficiency in the plasma protein Ceruloplasmin, crucial for iron transport within the body. The absence or lack of this protein leads to iron accumulation in vital organs such as the brain, liver, and pancreas, ultimately resulting in severe complications including neurological symptoms, diabetes, anemia, and retinal degeneration.

The granting of ODD is a pivotal milestone that illustrates Kedrion’s commitment to addressing some of the most pressing medical needs in the realm of rare and ultra-rare diseases. Kedrion has exemplified ingenuity by transforming industrial plasma waste into potentially life-saving treatments. This innovative approach not only repurposes unused plasma fractionation intermediates but also underscores a shift towards sustainability in biopharmaceutical development.

Innovative Approach to Therapy Development

Kedrion's Chief R&D and Innovation Officer, Andrea Caricasole, noted the importance of this designation as a validation of their scientific initiatives targeting rare conditions. The treatment leverages the therapeutic potential of Ceruloplasmin, aiming to provide new options for patients suffering from an ultra-rare, neurodegenerative disorder that presently lacks effective treatment pathways. Caricasole emphasized the transformative implications of this milestone for patients who currently have no approved drug for Aceruloplasminemia.

Collaboration and Research Innovations

The road to this designation has been shaped by collaborative efforts alongside academic and clinical partners, further backed by financial support from the Italian Ministry for Enterprises and Made in Italy (MIMIT). This support is channeled through the