Portola Pharmaceuticals, Inc.® (Nasdaq: PTLA) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion on the marketing authorization application (MAA) for Ondexxya™ (andexanet alfa). The Committee has recommended that the European Commission (EC) grant Ondexxya conditional approval for the reversal of the anticoagulant effects of the Factor Xa inhibitors apixaban or rivaroxaban in patients experiencing uncontrolled or life-threatening bleeding.
The MAA will now be reviewed by the EC, which has the authority to approve medicines for use in the 28 countries of the European Union, Norway, Liechtenstein and Iceland. If approved, Ondexxya would be the first and only specific reversal agent available in Europe to address life-threatening bleeding associated with the use of apixaban or rivaroxaban. The EC is expected to issue a decision in early May 2019.
«Today’s positive CHMP opinion represents a significant step forward in patient care and in the ability of clinicians to address life-threatening bleeds associated with the use of Factor Xa inhibitors,» said Jan Beyer-Westendorf, M.D., Head of the Thrombosis Research Unit, Department of Medicine I; Division Hematology, University Hospital Dresden, Germany. «Given the number of patients currently taking Factor Xa inhibitors and the anticipated growth of their utilization in Europe, the medical community has been eagerly awaiting the availability of an antidote that, in case of emergencies, can rapidly reverse the anticoagulating effects of rivaroxaban or apixaban. The European Society of Cardiology has already recognized the potential role of andexanet alfa, and we look forward to the potential approval and availability of this important medicine.»
The MAA submission is based on data from two Phase 3 ANNEXA studies that evaluated the safety and efficacy of Ondexxya in reversing the anticoagulant activity of the Factor Xa inhibitors rivaroxaban or apixaban in healthy subjects. Results of the ANNEXA studies were published in 2015 in The New England Journal of Medicine. The MAA also included adjudicated efficacy and safety data from bleeding patients enrolled in ANNEXA-4, the Company’s Phase 3b/4 study, the full results of which were published in The New England Journal of Medicine in February 2019.
“Today’s opinion is an important milestone for Portola and for patients in Europe, where the use of Factor Xa inhibitors is higher than in the U.S.,” said Scott Garland, Portola’s president and chief executive officer. “Given the consistency of the ANNEXA-4 data and the significant unmet need for a Factor Xa inhibitor reversal agent, we believe Ondexxya has the potential to benefit thousands of patients and we look forward to the opportunity to expand patient access in Europe this year.”
For a conditional approval, Portola is required to provide final study reports for both the ANNEXA-4 trial and the randomized controlled clinical trial requested by the U.S. Food and Drug Administration (FDA), as well as additional pharmacokinetic data.
Andexanet alfa was approved by the FDA in May 2018 and is marketed by Portola in the U.S. under the trade name Andexxa® [coagulation factor Xa (recombinant), inactivated-zhzo].
About Portola Pharmaceuticals, Inc.
Portola Pharmaceuticals is a commercial-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapeutics that could significantly advance the fields of thrombosis and other hematologic diseases. The Company’s two FDA-approved medicines are Andexxa® [coagulation factor Xa (recombinant), inactivated-zhzo], the first and only antidote for patients treated with rivaroxaban or apixaban when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding, and Bevyxxa®(betrixaban), the first and only oral, once-daily Factor Xa inhibitor for the prevention of VTE in adult patients hospitalized for an acute medical illness. The company also is advancing cerdulatinib, a Syk/JAK inhibitor for the treatment of hematologic cancers.
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 the regulatory pathway for potential approval of Ondexxya in the EU. Risks that contribute to the uncertain nature of the forward-looking statements include: the risk that the EMA may not approve Ondexxya in the currently anticipated timelines or at all, and that any marketing approvals or reimbursement limitations may have significant limitations on its use; the risk that physicians, patients and payers may not see the benefits of utilizing Andexxa or Bevyxxa for the indications which they are approved; our ability to continue to manufacture our products and to expand approved manufacturing facilities; the possibility of unfavorable results from additional clinical trials involving Andexxa; the risk that Portola may not obtain additional regulatory approvals necessary to expand approved indications for Andexxa; our expectation that we will incur losses for the foreseeable future and will need additional funds to finance our operations; the accuracy of our estimates regarding expenses and capital requirements; our ability to successfully build a hospital-based sales force and commercial infrastructure; our ability to obtain and maintain intellectual property protection for our product candidates; and our ability to retain key scientific or management personnel. These and other risks and uncertainties are described more fully in our most recent filings with the Securities and Exchange Commission, including our most recent quarterly report on Form 10-Q. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.