Kedrion receives FDA Orphan Drug Designation for investigational plasma-derived treatment for Congenital Aceruloplasminemia

FORT LEE, N.J., Aug. 7, 2025 /PRNewswire/ -- Kedrion is proud to announce that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to investigational treatment for Congenital Aceruloplasminemia, a rare genetic disorder affecting iron metabolism.

This designation marks a significant milestone in Kedrion's commitment to addressing the unmet medical needs of patients living with ultra-rare diseases and exemplifies sustainable innovation in plasma-derived therapies.

About Congenital Aceruloplasminemia

Aceruloplasminemia is an autosomal recessive disorder caused by mutations in the CP gene, leading to a deficiency or absence of ceruloplasmin, a protein essential for iron transport. The condition results in iron accumulation in the brain, liver, and pancreas, causing a range of symptoms including:

• Neurological issues: tremors, ataxia, dystonia
• Psychiatric symptoms: depression, cognitive decline
• Systemic effects: diabetes mellitus, anemia, retinal degeneration

Due to its rarity and complexity, Aceruloplasminemia is often misdiagnosed, delaying access to care and treatment.

Source: GARD (Genetic and Rare Diseases Information Center) – https://www.rarediseases.info.nih.gov/diseases/9499/aceruloplasminemia

A key step towards therapeutic innovation and sustainability

The FDA's Orphan Drug Designation recognizes the potential of Kedrion's plasma-derived ceruloplasmin to address an unmet need in treatment. This achievement is particularly significant because Kedrion is using industrial plasma processing waste to extract and purify new candidate therapeutic proteins.

This milestone builds on Kedrion's ongoing research efforts aimed at understanding ceruloplasmin biology and Aceruloplasminemia mechanisms, including two recent publications conducted in collaboration with a network of academic institutions, clinical research centers, and national biomedical organizations.

"Receiving Orphan Drug Designation from the FDA is a fundamental step in our journey to bring hope to patients affected by Congenital Aceruloplasminemia," said Andrea Caricasole, Chief Research and Innovation Officer at Kedrion. For Kedrion, this designation reinforces our long-term commitment to innovation in the field of ultra-rare diseases and is in line with our strategy of systematically leveraging unused plasma fractions to develop novel therapies to maximize the value of donated plasma, a precious and limited resource. We believe this progress will inspire further discoveries and collaborations, ultimately expanding the horizon of possibilities for patients who have long been underserved."

Looking ahead

Kedrion is committed to advance plasma-derived therapies and to support patients with rare and ultra-rare conditions. The Orphan Drug Designation for ceruloplasmin is a testament to the power of scientific collaboration, sustainable innovation, and the importance of advocacy in rare disease communities.

About Kedrion Biopharma

Kedrion Biopharma collects and fractionates blood plasma to produce and distribute plasma-derived therapies for rare, ultra-rare, and debilitating conditions like Coagulation and Neurological Disorders, Immunodeficiencies, and Rh sensitization. We employ approximately 5,200 people worldwide and our industrial network includes plasma collection centers in the United States and in the Czech Republic, and production facilities across 5 countries. We are committed to creating a world where science and care know no bounds, partnering with the medical-scientific community, institutions, patient advocacy groups, and research bodies to foster innovation and improve care. Every connection we make impacts someone, somewhere.

Contacts: Jennifer Donegan – US Communications Lead // [email protected]

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SOURCE Kedrion Biopharma