Kedrion Biopharma Receives Orphan Drug Designation for COAGADEX® for Acquired Factor X Deficiency (aFXD) and Starts Clinical Trial to Evaluate Efficacy and Safety

FORT LEE, N.J., July 29, 2025 /PRNewswire/ -- Kedrion Biopharma is proud to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Coagulation Factor X human (tradename COAGADEX) for the treatment of acquired Factor X Deficiency (aFXD), a rare hematologic disorder with a global prevalence of < 1 in 1,000,000 people¹. In addition, the FDA has provided a "Study may proceed letter" for the initiation of a clinical trial to evaluate the efficacy and safety of Coagadex in treating active bleeding episodes and managing peri-operative bleeding in patients with aFXD associated with AL amyloidosis.

COAGADEX is a plasma-derived human coagulation factor concentrate. It is currently approved in 38 countries for treatment of Hereditary Factor X deficiency in adults and children. It is specifically indicated for routine prophylaxis to reduce the frequency of bleeding episodes, on-demand treatment and control of bleeding episodes and perioperative management of bleeding in patients with mild, moderate, and severe hereditary Factor X deficiency. Important Safety Information for COAGADEX is provided at the end of this release.

"Receiving orphan drug designation for COAGADEX in aFXD is a significant milestone for Kedrion," said Bob Rossilli, Chief Commercial Officer and US General Manager at Kedrion Biopharma. "This designation underscores the unmet need for therapies to treat aFXD and the potential of COAGADEX in this space. Kedrion is committed to advancing care for patients with rare conditions, and we look forward to seeing the results of this clinical study."

The most common cause of acquired FX (aFX) deficiency is AL amyloidosis. —Amyloid fibrils bind to and reduce circulating Factor X levels, shortening its half-life and significantly increasing the risk of bleeding. A clinical trial aimed at addressing this treatment gap has commenced to evaluate COAGADEX's ability to restore hemostasis in this vulnerable population, and patient recruitment is currently ongoing.

The first trial site is being activated and will focus on both moderate and severe forms of aFXD, defined by Factor X coagulant activity (FX:C) levels below 50% of normal. 

"This study represents a pivotal step in evaluating the therapeutic potential of COAGADEX beyond HFXD." said Nisha Jain, VP Global Clinical Development and Strategy. "By targeting aFXD in patients with light chain amyloidosis, we are advancing our commitment to precision treatment in rare bleeding disorders. The orphan drug designation and the FDA's green light for this trial validates the urgency and potential of our work."

For more information about the trial, visit Study Details | A Study Investigating Coagadex in the Treatment AFXD Associated With AL Amyloidosis | ClinicalTrials.gov

About COAGADEX

COAGADEX is currently approved for the treatment and perioperative management of bleeding episodes in patients with hereditary Factor X deficiency. It is manufactured by Bio Products Laboratory Limited and distributed in the United States by Kedrion Biopharma Inc. 

For more information about COAGADEX visit: www.coagadex.com

Kedrion Biopharma is a biopharmaceutical company that collects and fractionates blood plasma to produce and distribute worldwide plasma-derived therapies for use in treating and preventing rare and debilitating conditions like Coagulation and Neurological Disorders, Primary Immunodeficiencies, and Rh sensitization, which can lead to Hemolytic Disease of the Fetus and Newborn. Kedrion is a global player in plasma derivatives and rare disease medicines, employing over 5,200 people worldwide.

¹Orphanet: Prevalence_of_rare_diseases_by_decreasing_prevalence_or_cases.pdf

Important Safety Information for COAGADEX

Allergic type hypersensitivity reactions, including anaphylaxis, are possible with COAGADEX. If symptoms occur, patients should discontinue use of the product immediately, contact their physician, and administer appropriate treatment.

The formation of neutralizing antibodies (inhibitors) to factor X is a possible complication in the management of individuals with factor X deficiency. Carefully monitor patients taking COAGADEX for the development of inhibitors by appropriate clinical observations and laboratory tests.

COAGADEX is made from human plasma and may contain infectious agents, e.g. viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent. No cases of transmission of viral diseases, vCJD or CJD, have been associated with the use of COAGADEX.

In clinical studies, the most common adverse reactions (frequency ≥5% of subjects) with COAGADEX were infusion site erythema, infusion site pain, fatigue and back pain.

Media Contact: Jennifer Donegan, US Communications Lead
[email protected] 

SOURCE Kedrion Biopharma