Kedrion Biopharma, an Italy-based biopharmaceutical company, announced on Thursday that it has received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) for its investigational treatment for Congenital Aceruloplasminemia (ACP), a rare genetic disorder of iron metabolism.

Kedrion produces and distributes plasma-derived therapies for rare, ultra-rare, and debilitating conditions like Coagulation and Neurological Disorders, Immunodeficiencies, and Rh sensitisation. Aceruloplasminemia 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.

This milestone is the result of collaborative efforts with academic and clinical partners and is supported by the Italian Ministry for Enterprises and Made in Italy (MIMIT) through the research project named 'NATURAL', which aim to foster research for new therapies by leveraging unused plasma fractionation intermediates.

Kedrion says that it will continue to advance this treatment for Aceruloplasminemia toward clinical development in Europe, with the goal of enabling timely access for patients who currently have no effective treatment options.