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Portola Pharmaceuticals Announces FDA Accepts New Drug Application for Priority Review and EMA Validates Marketing Authorization Application for Oral, Factor Xa Inhibitor Anticoagulant Betrixaban
Additionally, Portola announced that the
"With the filing of our betrixaban NDA and MAA validation, we now look forward to working with the FDA and EMA with the potential for betrixaban to be the first anticoagulant approved for in-hospital and extended-duration VTE prophylaxis in high-risk acute medically ill patients," said Bill Lis, chief executive officer of Portola.
The NDA and MAA for betrixaban are supported by data from Portola's 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. Full results from the multicenter, randomized, active-controlled APEX Study were presented at the 62nd Annual
About VTE in Acute Medically Ill Patients
Acute medically ill patients are those hospitalized for serious, common medical conditions, including heart failure, stroke, infection and pulmonary disease. Because of their underlying disorder or immobilization during hospitalization, they are at increased risk of VTE, a serious and potentially life-threatening blood clot (thrombus). VTE, which includes both deep vein thrombosis (DVT) and pulmonary embolism (PE), is a major cause of preventable morbidity and mortality and re-hospitalization in the acute medically ill patient population.
In the G7 countries, an estimated 24 million acute medically ill patients are hospitalized each year and are at risk of VTE, either while in the hospital or following discharge. More than 1 million VTE events and 150,000 VTE-related deaths occur annually in acute medically ill patients in the G7 countries, despite the standard use of injectable enoxaparin and other heparins in the hospital. More than half of VTE events occur after the patient is discharged. However, no anticoagulant, including enoxaparin or any of the marketed oral Factor Xa inhibitors, is approved for in-hospital and extended-duration VTE prophylaxis in acute medically ill patients who are hospitalized.
Betrixaban, an investigational drug, directly inhibits the activity of Factor Xa, an important validated target in the blood coagulation pathway, to prevent life-threatening thrombosis. Betrixaban has distinct properties that may allow it to demonstrate clinical benefit without the significant imbalance in the risk of major bleeding seen with other agents in the class. These include a 19-25-hour half-life for once-daily dosing; a low peak-to-trough drug concentration ratio that minimizes anticoagulant variability; low renal clearance; and no significant CYP3A4 metabolism, which may reduce the risk of drug-drug interactions.
About Portola Pharmaceuticals, Inc.
Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding development of our product candidates, our regulatory applications and estimated timelines associated therewith. Risks that contribute to the uncertain nature of the forward-looking statements include: failure to obtain regulatory approval for one or more of our product candidates, our expectation that we will incur losses for the foreseeable future and will need additional funds to finance our operations; the results of our clinical trials related to the efficacy and safety of our product candidates; our potential inability to manufacture our product candidates on a commercial scale in a timely or cost-efficient manner; the accuracy of our estimates regarding expenses and capital requirements; regulatory developments in
i Cohen AT, Harrington RA, Goldhaber SZ, Hull RD, Wiens BL, et al. Extended thromboprophylaxis with betrixaban in acutely ill medical patients. N Engl J Med. 2016;375:534-544.
ii Gibson CM, Chi G, Halaby R, Korjian S, Daaboul Y, et al. Extended-Duration Betrixaban Reduces the Risk of Stroke Versus Standard-Dose Enoxaparin Among Hospitalized Medically Ill Patients: An APEX Trial Substudy (Acute Medically Ill Venous Thromboembolism Prevention With Extended Duration Betrixaban). Circulation. https://doi.org/10.1161/CIRCULATIONAHA.116.025427.