Portola Pharmaceuticals Inc. announced that it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking approval to market betrixaban for extended-duration prophylaxis of venous thromboembolism (VTE) in acute medically ill patients with risk factors for VTE. Betrixaban, an FDA Fast Track-designated investigational drug, is an oral, once-daily Factor Xa inhibitor anticoagulant.
“Hospitalized acute medically ill patients are at significant risk of experiencing a potentially life-threatening VTE event, both during their stay and after discharge, especially within the first four weeks. Yet, no drug is approved in the United States for extended use to prevent VTE in these patients. We are committed to bringing betrixaban to market to address this urgent unmet medical need,” said John Curnutte, M.D. Ph.D., executive vice president, research and development at Portola. “If approved, betrixaban would be the first anticoagulant indicated for the prevention of VTE in acute medically ill patients both during hospitalization and for an extended period after the patient returns home.”
The NDA for betrixaban is supported by data from the pivotal Phase 3 APEX Study, which enrolled 7,513 patients at more than 450 clinical sites worldwide and assessed the superiority of extended-duration anticoagulation with oral betrixaban for 35 - 42 days compared with standard-duration injectable enoxaparin for 10+4 days in preventing VTE in high-risk acute medically ill patients. Results showed that betrixaban reduced the incidence of VTE compared with enoxaparin at a p value approaching statistical significance (p=0.054) in the primary efficacy analysis subgroup of 3,870 patients with elevated D-dimer levels. It also significantly reduced VTE in several pre-specified analyses of the primary efficacy analysis subgroup as well as in the overall study population (p=0.006) of 7,513 patients. No statistical difference in major bleeding was observed between the betrixaban and enoxaparin arms in either of the primary analysis patient subgroup or in the overall study population.
“The submission of the betrixaban NDA as planned is an important accomplishment for Portola and our regulatory and clinical teams as a first step towards a potential approval in 2017,” said Bill Lis, chief executive officer of Portola. “Based on the totality of data from the pivotal Phase 3 APEX study, there is strong support among the medical community for the use of betrixaban in preventing VTE in medically ill patients. We believe our dossier with the APEX Study as the basis shows clear evidence of betrixaban’s efficacy and safety. We look forward to working with the FDA as they review our application.”
Portola expects a response from the FDA within 60 days as to whether the NDA is complete and acceptable for filing. The Company plans to submit a Marketing Authorization Application for approval of betrixaban in the EU by the end of this year.
