Kedrion’s COAGADEX® Gets FDA Orphan Drug Status for Rare Bleeding Disorder

Kedrion’s COAGADEX® Received Confirmation of ‘Orphan Drug Designation'

Announcement

Kedrion Biopharma’s COAGADEX is now declared an orphan drug designation from the US Food and Drug Administration (FDA). The coagulation factor X human (tradename COAGADEX) will be a treatment for factor X deficiency (afxd), a rare hematologic disorder with a worldwide prevalence of <1 in 1,000,000 individuals. The FDA has issued a ‘study may proceed letter’ for starting with the clinical trial to evaluate safety and efficacy measures of Coagadex in managing peri-operative bleeding in patients with AL amyloidosis and to treat active bleeding activities. This new orphan drug designation is an additional effort in global drug development.

Coagadex and Causes of aFX

Coagadex is a plasma-driven human coagulation factor concentrate. It recently received approval in around 38 countries for hereditary factor X deficiency treatment in children and adults. It is designed for routine prophylaxis to diminish the excessive bleeding episodes, to control perioperative management bleeding, and to provide on-demand treatment for mild, moderate, and extreme hereditary factor X deficiency in patients. Bio Products Laboratory Limited manufactured Coagadex, and the United States, by Kedrion Biopharma Inc., was a distribution channel for the same.

The most obvious cause of acquired FX (afx) deficiency is AL amyloidosis. The amyloid fibrils limit the circulation of factor X levels, shorten lifespan, and elevate the risk of bleeding. The clinical trial is focused on identifying the treatment bridge that has nearly will be commenced to evaluate Coagadex’s potential to restore hemostasis among vulnerable groups, and the patient trial is currently in process.

Views and Statements

Chief Commercial Officer and US general manager at Kedrion Biopharma, Bob Rossilli, said, “The orphan drug designation for COAGADEX in Afxd is a prominent milestone for the company. This designation holds a primary position in discovering unmet needs for therapies for afxd and demonstrates the potential of COAGADEX in this area. The company is committed to providing advanced care for patients with rare diseases. Now we have eyes on the results of the clinical study.”

VP Global Clinical Development and Strategy, Nisha Jain, said, “This study is a vital step towards evaluation of the therapeutic potential of coagadex beyond HFXD. By closely focusing on afxd in patients with limited chain to amyloidosis, it represents our dedication to precision treatment in rare bleeding disorders. This designation and FDA’s thumbs up for this trial indicate the emergency and capability of our work to introduce best-in-class therapies.”