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Kedrion Biopharma Achieves Milestone with COAGADEX®

Kedrion Biopharma has reached an important milestone in the pharmaceutical landscape as the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Coagulation Factor X, marketed as COAGADEX®, specifically for the treatment of acquired Factor X Deficiency (aFXD). This rare blood disorder affects fewer than one in a million individuals worldwide, underscoring the significance of this designation. Furthermore, the FDA has approved the initiation of a clinical trial aimed at assessing the efficacy and safety of COAGADEX® in managing active bleeding episodes and facilitating peri-operative bleeding management in patients who have aFXD linked to AL amyloidosis.

COAGADEX® is derived from human plasma and serves as a coagulation factor concentrate. It has existing approvals in 38 countries for treating hereditary Factor X deficiency in both adults and children. The indication covers routine prophylaxis to lessen the frequency of bleeding incidents, immediate treatment for bleeding episodes, and managing bleeding during surgical procedures for patients across various severities of hereditary Factor X deficiency. Kedrion Biopharma highlights this recent advancement as a cornerstone in their commitment to offering groundbreaking therapies for rare diseases and conditions.

Bob Rossilli, Kedrion's Chief Commercial Officer and US General Manager, emphasized the importance of this acknowledgment, stating, "Receiving orphan drug designation for COAGADEX in aFXD is a significant milestone for Kedrion. 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." This trial aims to fill a treatment void for patients suffering from this rare disorder, where AL amyloidosis is a leading cause of acquired FX deficiency. In such cases, amyloid fibrils bind to circulating Factor X, leading to a reduction in its levels and significantly amplifying the risk of bleeding.

The clinical trial is currently underway, with patient recruitment actively ongoing. The initial trial site focuses on assessing moderate and severe cases defined by Factor X coagulant activity (FXC) levels below 50% of normal levels. Nisha Jain, VP of Global Clinical Development and Strategy at Kedrion, remarked, "This study represents a pivotal step in evaluating the therapeutic potential of COAGADEX beyond hereditary Factor X deficiency. By targeting acquired Factor X deficiency in patients with light chain amyloidosis, we are advancing our commitment to precision treatment in rare bleeding disorders.”

COAGADEX® has a compelling history; it is currently dispensed in the U.S. by Kedrion Biopharma and manufactured by Bio Products Laboratory Limited. It is praised for its capacity to manage and mitigate bleeding disorders, making it a vital resource for patients with hereditary deficiencies.

For those interested in learning more about the clinical trial of COAGADEX®, further information can be found at ClinicalTrials.gov.

As Kedrion Biopharma continues innovating and providing solutions for blood disorder treatments, their ongoing efforts forge a hopeful path for individuals with rare conditions like aFXD. The company employs over 5,200 personnel globally, reinforcing its role as a significant player in the realm of plasma-derived therapies dedicated to rare and debilitating medical conditions.