Portola Pharmaceuticals announces validation of MAA by EMA for IndexXa a Factor Xa inhibitoraAntidote

EMA's CHMP to begin centralized review procedure.

“An estimated 73,000 patients treated with an oral Factor Xa inhibitor will be hospitalized this year due to major bleeding or because they require urgent surgery in the EU5i. These patients are critically ill and could potentially benefit from a Factor Xa inhibitor antidote,” said Bill Lis, CEO of Portola. “As there is currently no approved antidote for Factor Xa inhibitors in the EU, we are working diligently to bring IndexXa to patients. The MAA validation is an important milestone toward achieving that goal.”

Portola’s MAA submission is based on data from two Phase III ANNEXA studies that evaluated the safety and efficacy of andexanet alfa in reversing the anticoagulant activity of the Factor Xa inhibitors rivaroxaban and apixaban in healthy volunteers. Results of the ANNEXA studies were published by The New England Journal of Medicine. The MAA also included limited adjudicated efficacy and safety data from initial patients enrolled in the ongoing ANNEXA-4 study. Portola is currently evaluating andexanet alfa in this global, Phase IIIb/IV single-arm, open-label study in patients receiving apixaban, rivaroxaban, edoxaban or enoxaparin who present with an acute major bleed. The study is enrolling patients in the United States, Canada and the EU.

On 17 August 2016, Portola received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for AndexXa. The company plans to meet with the FDA as soon as possible to resolve the outstanding questions in the CRL and determine appropriate next steps.