FORT LEE, N.J., July 29, 2025 — Kedrion Biopharma has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Coagulation Factor X human (marketed as COAGADEX) for treating acquired Factor X Deficiency (aFXD), a rare blood disorder affecting fewer than 1 in 1,000,000 individuals globally¹. Furthermore, the FDA has issued a “Study may proceed letter,” allowing a clinical trial to begin. This trial will assess Coagadex’s effectiveness and safety in managing active bleeding episodes and peri-operative bleeding for patients with aFXD linked to AL amyloidosis.

COAGADEX is a human coagulation factor concentrate derived from plasma. It is presently approved in 38 countries for addressing Hereditary Factor X deficiency in both adults and children. Its specific indications include routine preventative treatment to lessen the occurrence of bleeding, on-demand therapy and control for bleeding episodes, and managing bleeding around surgery for patients with mild, moderate, and severe hereditary Factor X deficiency. Crucial Safety Information for COAGADEX can be found at the conclusion of this press release.

“Attaining orphan drug designation for COAGADEX in aFXD represents a major achievement for Kedrion,” stated Bob Rossilli, Kedrion Biopharma’s Chief Commercial Officer and US General Manager. “This designation highlights the current lack of treatments for aFXD and COAGADEX’s potential role in this area. Kedrion is dedicated to improving care for individuals with rare disorders, and we are eager to see the outcomes of this clinical study.“

AL amyloidosis is the primary cause of acquired FX (aFX) deficiency. Amyloid fibrils attach to and lower circulating Factor X levels, reducing its lifespan and considerably elevating the risk of bleeding. A clinical trial has begun to bridge this treatment void, assessing COAGADEX’s capacity to reinstate hemostasis in this susceptible patient group, and patient enrollment is presently underway.

The initial trial site is now active and will concentrate on both moderate and severe forms of aFXD, characterized by Factor X coagulant activity (FX:C) levels under 50% of the normal range. 

“This investigation marks a crucial advancement in exploring COAGADEX’s therapeutic capabilities beyond HFXD,” commented Nisha Jain, VP Global Clinical Development and Strategy. “By addressing aFXD in individuals with light chain amyloidosis, we are furthering our dedication to precise treatment for rare bleeding conditions. Both the orphan drug designation and the FDA’s approval for this trial affirm the importance and promise of our efforts.”

To learn more about the trial, please visit

About COAGADEX

COAGADEX is presently approved for managing bleeding episodes and perioperative care in patients with hereditary Factor X deficiency. Bio Products Laboratory Limited manufactures it, and Kedrion Biopharma Inc. distributes it within the United States. 

For additional details about COAGADEX, visit:

Kedrion Biopharma is a biopharmaceutical firm that gathers and processes blood plasma to create and globally distribute plasma-derived treatments. These therapies are used for managing and preventing rare and severe conditions such as Coagulation and Neurological Disorders, Primary Immunodeficiencies, and Rh sensitization, which can result in Hemolytic Disease of the Fetus and Newborn. Kedrion is a worldwide leader in plasma derivatives and rare disease medications, with a global workforce exceeding 5,200 employees.

¹Orphanet:

Important Safety Information for COAGADEX

Allergic hypersensitivity reactions, including severe anaphylaxis, may occur with COAGADEX. Should symptoms arise, patients must stop using the product immediately, reach out to their doctor, and receive suitable treatment.

The development of neutralizing antibodies (inhibitors) against factor X is a potential complication in managing individuals with factor X deficiency. Patients receiving COAGADEX should be closely monitored for inhibitor development through relevant clinical observations and laboratory tests.

COAGADEX is derived from human plasma and could potentially contain infectious agents, such as viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent, and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. No instances of viral disease, vCJD, or CJD transmission have been linked to COAGADEX use.

During clinical trials, the most frequently observed adverse reactions (occurring in ≥5% of subjects) with COAGADEX included redness at the infusion site, pain at the infusion site, tiredness, and back discomfort.

Media Contact: Jennifer Donegan, US Communications Lead
 

SOURCE Kedrion Biopharma