Novoseven off-label – pro
Recombinant activated factor seven (VIIa) is a vitamin K-dependent glycoprotein that is structurally similar to human plasma-derived factor VIIa. It has received approval from the Food and Drug Administration (FDA) for the treatment of bleeding episodes in patients with hemophilia A or B who have developed inhibitors to factor VIII or factor IX, respectively. It has recently been approved in the EU for Glantzman’s thromabasthenia. However, because of its effectiveness in pro-coagulation, it can potentially be used for a wide variety of bleeding situations. Coupled with its high cost, this caused a need for standards. The unlabeled uses of factor VIIa (recombinant) are constrained by high costs and limited clinical efficacy data. Currently, there are no national guidelines for the unlabeled indications of factor VIIa (recombinant); many institutions have written and follow their own institutional guidelines. Because of the lack of data, the approach of setting up intitutional gatekeepers to evaluate specific cases, has become widespread. Usually, an expert in each of the specialties of trauma, neurosurgery, hepatology and hematology are appointed to approve use in specific cases.
According to the FDA, Novoseven has been linked to heart attacks, strokes, deaths, and other health complications in patients given the medicine for other types of out-of-control bleeding, such as cerebral hemorrhages. The drug has shown promise as a treatment for cerebral hemorrhages, which is a potentially deadly kind of stroke caused by bleeding in the brain. The majority of the reported complications associated with the drug involved off-label use, instances where the drug was used to treat a condition not specifically approved by the FDA. Novo Nordisk changed the warning in the package insert to include information on side-effects in patients who do not have hemophilia. Novo is currently conducting studies on the safety of the drug in such patients.
NovoSeven (coagulation factor VIIa [recombinant]) package insert. Princeton, NJ: Novo Nordisk; 2005 Jun.
Lam MS, Sims-McCallum RP. Recombinant factor VIIa in the treatment of nonhemophiliac bleeding. Ann Pharmacother. 2005; 39:885–91.
Friederich PW, Henny CP, Messelink EJ et al. Effect of recombinant activated factor VII on perioperative blood loss in patients undergoing retropubic prostatectomy: a double-blind placebo-controlled randomised trial. Lancet. 2003; 361:201–5.
Celeste N. Rudisill; Rebecca H. Hockman; Kathlene A. Degregory; Alan H. Mutnick; Barbara Gail Macik Implementing Guidelines for the Institutional Use of Factor VIIa (Recombinant): A Multidisciplinary Solution Am J Health-Syst Pharm. 2006;63(17):1641-1646