Kedrion Biopharma has announced that its human coagulation factor X concentrate, COAGADEX®, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of acquired factor X deficiency (AFXD), a rare hematologic disorder affecting fewer than one in a million individuals globally.

The FDA has also issued a “study may proceed” letter, clearing the way for a clinical trial to evaluate the safety and efficacy of COAGADEX in managing peri-operative bleeding and treating active bleeding episodes in patients with AL amyloidosis a key cause of AFXD. The trial aims to assess COAGADEX’s potential in restoring hemostasis in vulnerable patient populations.

COAGADEX is a plasma-derived human coagulation factor concentrate that has already received approval in approximately 38 countries for the treatment of hereditary factor X deficiency (HFXD) in both adults and children. It is used for routine prophylaxis, on-demand bleeding treatment, and perioperative bleeding management in mild to severe cases. While COAGADEX is manufactured by Bio Products Laboratory Limited, its distribution in the United States is handled by Kedrion Biopharma Inc.

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Commenting on the FDA designation, Bob Rossilli, Chief Commercial Officer and U.S. General Manager at Kedrion Biopharma, stated, “The orphan drug designation for COAGADEX in AFXD is a significant milestone. It highlights the urgent need for new therapies in this underserved area and underscores our commitment to advancing care for patients with rare diseases. We now look forward to the results of the clinical trial.”

Dr. Nisha Jain, VP of Global Clinical Development and Strategy at Kedrion, added, “This study marks a critical step in exploring COAGADEX’s broader therapeutic potential. By focusing on AFXD linked to amyloidosis, we are reinforcing our dedication to precision medicine in rare bleeding disorders. The FDA’s support validates the importance of our mission.”

The new designation represents a key advancement in global rare disease treatment efforts and expands the potential reach of COAGADEX in the management of bleeding disorders beyond hereditary indications.

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