Therapy Specific to Hereditary Factor X Deficiency Approved by FDA
FDA on Tuesday announced the approval of human plasma–derived coagulation factor X as a replacement therapy for patients 12 years of age or older with hereditary factor X deficiency, a rare disorder.
The agency said the product is the first to specifically treat the bleeding episodes associated with hereditary factor X deficiency.
Known by the brand name Coagadex, the biologic is for use in the on-demand treatment and control of bleeding episodes in patients with hereditary factor X deficiency and perioperative management of bleeding in patients with the mild form of the disorder, FDA said.
The labeling for the product recommends, for the treatment of bleeding episodes in patients with the hereditary disorder, a dosage of 25 IU of factor X activity per kilogram of body weight, repeated every 24 hours until the bleeding stops.
For the perioperative management of bleeding in patients with mild hereditary factor X deficiency, the product's labeling recommends that sufficient coagulation factor X be administered preoperatively to raise the patient's plasma factor X concentration to 70–90 IU/dL. Postoperatively, the patient should receive enough coagulation factor X to maintain a plasma factor X concentration of at least 50 IU/dL until the person is not at risk of surgery-related bleeding.
All doses, the labeling states, are to be given by i.v. infusion at a rate of 10 mL/min.
Among the 18 patients with hereditary factor X deficiency who received at least one dose of the replacement therapy during either of its two clinical studies, the most common adverse reactions were infusion-site erythema, infusion-site pain, fatigue, and back pain, according to the labeling.
Coagadex will be available in kits containing a 250- or 500-IU single-use vial of lyophilized coagulation factor X, a 2.5- or 5-mL vial of sterile water for injection, and a vial-to-vial transfer device for use in reconstituting the freeze-dried biologic. The labeling details an eight-step process for reconstituting the product and transferring it to a syringe.
Once reconstituted, the product should be used within one hour.
Until reconstitution, the product and other components of the kit should be stored in the original package in a refrigerator or at room temperature.
The product will be distributed by Bio Products Laboratory USA Inc.
Its parent company, Bio Products Laboratory Ltd., said it expects for Coagadex to be available to U.S. patients “in December 2015 through a network of specialty pharmacies and authorized distributors.”