Kedrion Biopharma has announced a major step forward in rare disease treatment: its plasma-derived therapy, COAGADEX® (Coagulation Factor X, Human), has received Orphan Drug Designation (ODD) from the U.S. FDA for treating acquired Factor X deficiency (aFXD). This rare blood disorder affects fewer than 1 in 1,000,000 people globally. Alongside the designation, the FDA has also approved the initiation of a clinical trial to study COAGADEX's safety and effectiveness in aFXD patients, especially those with AL amyloidosis.
Expanding COAGADEX's Reach Beyond Hereditary Factor X Deficiency
COAGADEX is already approved in 38 countries to treat hereditary Factor X deficiency (HFXD) in both adults and children. It's used to prevent bleeding episodes, manage active bleeds, and control bleeding during surgeries. With this new ODD, Kedrion aims to expand COAGADEX's application to acquired forms of the deficiency, which are much rarer and harder to treat.
Bob Rossilli, Chief Commercial Officer at Kedrion Biopharma, emphasized the importance of this milestone: "This designation highlights the unmet medical need in aFXD and recognizes COAGADEX's potential to meet that need."
Understanding Acquired Factor X Deficiency and AL Amyloidosis
Unlike hereditary FX deficiency, acquired FX deficiency often develops due to another condition—most commonly AL amyloidosis. In this disease, abnormal protein deposits (amyloid fibrils) bind to Factor X in the bloodstream, lowering its levels and speeding up its breakdown. This leads to a high risk of spontaneous bleeding and complications during surgery.
To address this, Kedrion has launched a clinical trial to see if COAGADEX can restore proper blood clotting in this vulnerable group. The first trial site is already active and enrolling patients with moderate to severe aFXD, defined by FX activity levels under 50% of normal.
A Precision Approach to a Rare Disorder
Nisha Jain, Kedrion's VP of Global Clinical Development and Strategy, called the trial a “pivotal step” in evaluating COAGADEX's potential beyond hereditary cases. “By targeting aFXD in patients with light chain amyloidosis,” she explained, “we are furthering precision treatment for rare bleeding disorders.” She also noted that the FDA's fast response to the trial proposal underscores the importance of their work.
Important Safety Information About COAGADEX
Like all plasma-derived products, COAGADEX carries some risks. Allergic reactions, including anaphylaxis, may occur. Patients should stop treatment and seek medical help if such symptoms arise. There's also a possibility of developing neutralizing antibodies (inhibitors) against Factor X, which may reduce treatment effectiveness.
Although no infections have been reported, COAGADEX is made from human plasma and may carry a theoretical risk of virus or prion transmission, such as vCJD or CJD. In clinical studies, the most common side effects (seen in at least 5% of patients) included infusion site redness, pain, fatigue, and back pain.
About Kedrion Biopharma
Kedrion Biopharma is a global biopharmaceutical company focused on collecting and processing blood plasma to develop treatments for rare and serious conditions. These include bleeding disorders, neurological diseases, immune deficiencies, and Rh sensitization. With over 5,200 employees worldwide, Kedrion remains committed to advancing care for patients living with rare and life-altering diseases.
