Kedrion Biopharma has received Orphan Drug Designation from the U.S. FDA for COAGADEX®, a plasma-derived human coagulation factor concentrate, targeting Acquired Factor X Deficiency (aFXD), a rare condition with extremely low prevalence worldwide. Alongside this designation, the FDA has authorized the start of a clinical trial to evaluate the efficacy and safety of COAGADEX in treating bleeding episodes and managing surgical bleeding in patients with aFXD linked to AL amyloidosis. This move expands the drug's scope beyond hereditary Factor X deficiency (HFXD), where it is already approved in 38 countries.
About COAGADEX and Its Current Use
COAGADEX is currently indicated for patients with hereditary Factor X deficiency. It helps prevent and control bleeding episodes and supports patients undergoing surgery. The product is manufactured by Bio Products Laboratory Limited and distributed in the U.S. by Kedrion Biopharma. With established approval in multiple countries, COAGADEX plays a key role in the treatment of rare coagulation disorders.
Expanding to Treat Acquired Factor X Deficiency (aFXD)
The new focus is on acquired FX deficiency, most often caused by AL amyloidosis. In this condition, amyloid fibrils bind to Factor X, lowering its level in the blood and raising the risk of serious bleeding. Kedrion's upcoming clinical trial will target moderate and severe aFXD cases, defined as having Factor X activity below 50%. The trial aims to assess COAGADEX's potential to restore proper clotting in these high-risk patients.
Regulatory and Clinical Progress
The FDA's Orphan Drug Designation is a key milestone that highlights both the rarity of aFXD and the need for new treatment options. The green light for clinical trials signals regulatory support for further investigation into COAGADEX's expanded use. According to Kedrion executives, this study could redefine treatment standards for acquired bleeding disorders.
Safety Information and Monitoring
Like all plasma-derived therapies, COAGADEX carries certain risks. These include possible allergic reactions, formation of inhibitors (neutralizing antibodies), and theoretical risks of transmitting infectious agents. In clinical studies, common side effects included infusion site redness, pain, fatigue, and back pain. Ongoing patient monitoring is essential during treatment.
COAGADEX's expansion into aFXD represents a meaningful step in rare disease care. By addressing an urgent need in patients with amyloidosis-associated bleeding, Kedrion is working to offer a new option where few currently exist.
