Kedrion Biopharma has announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Coagulation Factor X human (Coagadex) for the treatment of acquired Factor X Deficiency (aFXD), a rare hematologic disorder with a global prevalence of < 1 in 1,000,000 people.
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.”
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.
