Kedrion announced that the FDA has granted Orphan Drug Designation to an 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.
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.
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."
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