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08 Aug 2025
Admin
News Article

Kedrion Receives FDA Orphan Drug Designation for Ceruloplasmin Therapy

Milestone in the treatment of ultra-rare disorder Congenital Aceruloplasminemia

FDA Recognition for Rare Disease Therapy

Kedrion Biopharma has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for its investigational treatment for Congenital Aceruloplasminemia — a rare genetic disorder affecting iron metabolism.

This designation reflects Kedrion’s commitment to ultra-rare disease innovation and its efforts to develop sustainable plasma-derived therapies.

About Congenital Aceruloplasminemia

Aceruloplasminemia is:

Genetic origin: Autosomal recessive disorder caused by mutations in the CP gene
Defining issue: Deficiency or absence of ceruloplasmin, a protein essential for iron transport
Impact: Abnormal iron accumulation in the brain, liver, and pancreas

Symptoms may include:

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

Due to its rarity and complex symptom profile, this condition is often misdiagnosed, delaying proper treatment.

Innovation Through Sustainable Science

The FDA’s Orphan Drug Designation acknowledges the therapeutic potential of Kedrion’s plasma-derived ceruloplasmin, which is being developed to address this unmet medical need.

What makes this project unique:

Utilizes industrial plasma processing waste to extract and purify new candidate therapeutic proteins
Advances circular innovation by making full use of plasma donations
Builds on two recent scientific publications in collaboration with universities, clinical research centers, and national biomedical bodies

Leadership Statement

“Receiving Orphan Drug Designation from the FDA is a fundamental step in our journey to bring hope to patients affected by Congenital Aceruloplasminemia.”

Andrea Caricasole, Chief Research and Innovation Officer, Kedrion

Caricasole added that this recognition strengthens Kedrion’s long-term innovation strategy in rare diseases while maximizing the value of donated plasma — a precious and limited resource.

Looking Ahead

Kedrion will continue to:

Advance plasma-derived therapy development
Collaborate with scientific and medical communities
Support rare and ultra-rare disease advocacy efforts

About Kedrion Biopharma

Core expertise: Collecting and fractionating human plasma to produce therapies for rare and debilitating conditions
Therapy focus: Coagulation disorders, neurological disorders, immunodeficiencies, and Rh sensitization
Global footprint:
- ~5,200 employees worldwide
- Plasma collection centers in the US and Czech Republic
- Manufacturing facilities in five countries

Partnerships: Works with institutions, patient groups, and research bodies to drive innovation and improve care

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