FDA Okays Coagadex for Rare Bleeding Disorder

Disclosures

October 20, 2015

The US Food and Drug Administration (FDA) today approved the first replacement therapy for hereditary Factor X deficiency, a rare bleeding disorder, the agency announced.

The new therapy, Coagulation Factor X (Coagadex, Bio Products Laboratory), is derived from human plasma. It is indicated for individuals aged 12 years or older with hereditary Factor X deficiency for on-demand treatment and control of bleeding episodes, and for perioperative management of bleeding in patients with a mild form of the condition.

A protein, Factor X turns on enzymes to help with normal blood clotting. Congenital Factor X deficiency, which causes blood not to clot properly, affects an estimated 1 in 500,000-1,000,000 individuals worldwide. The traditional treatment is either fresh frozen plasma or plasma-derived prothrombin complex concentrates.

The FDA evaluated the safety and efficacy of the Factor X replacement therapy in a multicenter, nonrandomized study involving 16 participants who were treated for spontaneous, traumatic, and heavy menstrual bleeding. The therapy effectively controlled bleeding in participants with moderate-to-severe cases of hereditary Factor X deficiency. It also controlled blood loss before and after surgery in five individuals with a mild deficiency. The agency did not identify any safety concerns.

The FDA designated the factor replacement therapy as an orphan drug, which gives manufacturers economic benefits that make commercializing a product for a small number of patients more feasible. The agency expedited approval by granting the drug both fast-track and priority-review status.

More information about today's announcement is available on the FDA website.

Comments

3090D553-9492-4563-8681-AD288FA52ACE
Comments on Medscape are moderated and should be professional in tone and on topic. You must declare any conflicts of interest related to your comments and responses. Please see our Commenting Guide for further information. We reserve the right to remove posts at our sole discretion.

processing....