Portola Pharmaceuticals Announces Validation of Marketing Authorization Application (MAA) by European Medicines Agency (EMA) ...
19 Août 2016 - 2:30PM
Portola Pharmaceuticals Inc.® (NASDAQ:PTLA) today announced that
the MAA for IndexXa™ (andexanet alfa; trade name in the United
States is AndexXa™), a Factor Xa inhibitor antidote, has been
submitted to the EMA, completed the validation period, and has been
accepted for review. IndexXa is in development for patients treated
with a Factor Xa inhibitor when reversal of anticoagulation is
needed due to life-threatening or uncontrolled bleeding and for
patients requiring urgent or emergency surgery. Portola is seeking
conditional approval of IndexXa in the EU through the centralized
procedure. With the acceptance of the application, the EMA’s
Committee for Medicinal Products for Human Use (CHMP) will now
begin the centralized review procedure under a standard 210-day
review period.
“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, chief executive officer 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 3
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 3b/4 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 August 17, 2016, Portola received a Complete Response Letter
(CRL) from the U.S. Food and Drug Administration (FDA) regarding
its Biologics License Application (BLA) for AndexXa. The Company
plans to meet with the FDA as soon as possible in order to resolve
the outstanding questions in the CRL and determine appropriate next
steps.
The Urgent Need for a Factor Xa Inhibitor
AntidoteAnnually, 1 to 4 percent of patients treated with
Factor Xa inhibitors may experience major bleeding, and an
additional one percent may require emergency surgery. Commensurate
with the increase in the use of Factor Xa inhibitors -- for stroke
prevention in atrial fibrillation; treatment and prevention of deep
vein thrombosis (DVT) and pulmonary embolism; and prevention of DVT
following knee or hip replacement surgery -- the number of hospital
admissions due to bleeding associated with these agents continues
to grow. In the EU5 an estimated 73,000 oral Factor Xa
inhibitor-treated patients will be hospitalized this year due to
major bleeding or will require urgent surgery.ii
About IndexXa IndexXa, an investigational drug,
is a modified human Factor Xa molecule that acts as a decoy to
target and sequester with high specificity both oral and injectable
Factor Xa inhibitors in the blood. Once bound, the Factor Xa
inhibitors are unable to bind to and inhibit native Factor Xa, thus
potentially allowing for the restoration of normal hemostatic
processes. IndexXa is the first compound being studied as an
antidote for Factor Xa inhibitors that directly and specifically
reverses anti-Factor Xa activity – the anticoagulant mechanism of
these agents.
About Portola Pharmaceuticals, Inc. Portola
Pharmaceuticals is a biopharmaceutical company developing product
candidates that could significantly advance the fields of
thrombosis and other hematologic diseases. The Company is advancing
three programs, including betrixaban, an oral, once-daily Factor Xa
inhibitor; IndexXa™ (andexanet alfa), a recombinant protein
designed to reverse the anticoagulant effect in patients treated
with an oral or injectable Factor Xa inhibitor; and cerdulatinib, a
Syk/JAK inhibitor in development to treat hematologic cancers.
Portola's partnered program is focused on developing selective Syk
inhibitors for inflammatory conditions. For more information, visit
www.portola.com and follow the Company on Twitter
@Portola_Pharma.
Forward-looking StatementsStatements 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 potential future regulatory
approval of andexanet alfa and patient populations that could
benefit from a Factor Xa inhibitor reversal agent. Risks that
contribute to the uncertain nature of the forward-looking
statements include the risk that we may be unable to satisfy
regulatory requirements for such approval, we may be unable to
manufacture andexanet alfa on a commercial scale and we will need
additional capital to fund our operations. 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, which was filed on
August 9, 2016. 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.
i France, Germany, Italy, Spain and the United
Kingdom
ii Portola estimate based on IMS reported oral Factor Xa
inhibitor sales in the EU5
Investor Contact:
Ana Kapor
Portola Pharmaceuticals
ir@portola.com
Media Contact:
Julie Normart
W2O Group
jnormart@w2ogroup.com
415.946.1087
Portola Pharmaceuticals (NASDAQ:PTLA)
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