EMA Grants Orphan Drug Designation to Kedrion's Treatment for ACP

The European Medicines Agency (EMA) has awarded Orphan Drug Designation to Kedrion's investigational treatment for Congenital Aceruloplasminemia (ACP), a rare genetic disorder affecting iron metabolism. ACP is characterized by a deficiency in Ceruloplasmin, leading to iron buildup in vital organs and severe symptoms. Kedrion's innovative approach repurposes unused plasma fractionation intermediates, targeting unmet medical needs in rare diseases.

This milestone underscores Kedrion's commitment to sustainable biopharmaceutical advances. Supported by the Italian Ministry for Enterprises and Made in Italy, this effort aligns with recent scientific findings in Nature Communications Biology and Lancet eBioMedicine, validating the potential of the treatment in addressing ACP's challenges.