Haemophilia is a congenital bleeding disorder occurres due to the dysfunction in or deficiency of coagulation factor VIII (Haemophilia A) or IX (Haemophilia B). People with hemophilia often bleed for longerperiods than others after an injury or a surgery. (1) Currently, the standard line of treatment for the disorder includes treatment with plasma-derived or recombinant concentrates of FVIII or FIX. (2) However, it is often seen that patients on the above treatment develop inhibitors to FVIII or FIX. Thus in this regard, it becomes essential to use a therapy that would help in the treatment of patients who develop inhibitors to FVIII or FIX.
FDA Approves Recombinant Human FVI For Haemophilia A or B
The USFDA has recently approved “Sevenfact” (recombinant analog of human FVII) for physicians in the treatment of Haemophilia. The medication bypasses factor VIII and Factor IX reactions, and promotes clot formation to control bleeding.
The approval of the drug (75 mcg/kg and 225 mcg/kg) was based on the positive result of clinical trials which accessed the safety, efficacy, and pharmacokinetics of the drug in participants with hemophilia A or B and inhibitors to FVIII or FIX. Headache, dizziness, infusion-related problems (reactions, site discomfort, and hematoma) and fever were some of the common side effects associated with Sevenfact. Additionally, the drug has been contraindicated for patients with allergy or hypersensitivity torabbits/rabbit proteins.
FDA had granted approval of drug to Laboratoire Francais du Fractionnement et des Biotechnologies S.A. (3)
1. Zimmerman B, Valentino LA. Hemophilia: in review. Pediatr Rev. 2013 Jul;34(7):289-94; 295.
2. Franchini M. The modern treatment of haemophilia: a narrative review. Blood Transfus. 2013;11(2):178–182.
3. FDA Approves Additional Treatment for Adults and Adolescents with Hemophilia A or B and Inhibitors. (11.04.20). FDA News Release. Retrieved from https://www.fda.gov/news-events/press-announcements/fda-approves-additional-treatment-adults-and-adolescents-hemophilia-or-b-and-inhibitors