Portola Pharmaceuticals Receives Complete Response Letter from FDA for Biologics License Application for AndexXa™ (andexane...
18 Août 2016 - 4:57AM
Portola Pharmaceuticals Inc.® (NASDAQ:PTLA) today announced that it
has received a Complete Response Letter (CRL) from the U.S. Food
and Drug Administration (FDA) regarding its Biologics License
Application (BLA) for AndexXa™ (andexanet alfa). An FDA-designated
Breakthrough Therapy, AndexXa is in development for patients
treated with a direct (apixaban, rivaroxaban, or edoxaban) or
indirect (enoxaparin) Factor Xa inhibitor when reversal of
anticoagulation is needed due to life-threatening or uncontrolled
bleeding. Currently, there is no FDA-approved antidote for
Factor Xa inhibitors.
In the CRL for AndexXa, the FDA requested that Portola provide
additional information primarily related to manufacturing. The
agency also asked for additional data to support inclusion of
edoxaban and enoxaparin in the label, and indicated it needs to
finalize its review of the clinical amendments to Portola’s
post-marketing commitments that recently were submitted.
“Because AndexXa addresses an urgent unmet medical need, we and
the FDA are committed to resolving the outstanding questions and
determining appropriate next steps. Portola’s goal is to define the
most expedient path to approval so we can meet the needs of these
patients who have no alternative,” said Bill Lis, chief executive
officer of Portola. “We plan to meet with the FDA as soon as
possible.”
Conference Call Details The Portola management
team will host a conference call and webcast tomorrow, Thursday,
August 18, 2016, at 8:30 am ET to provide more information about
AndexXa. The live call can be accessed by phone by calling 1 (844)
452-6828 (domestic) or 1 (765) 507-2588 (international) and
specifying conference call code 68482562. The webcast can be
accessed live on the Investor Relations section of the Company's
website at http://investors.portola.com. It will be
archived for 30 days following the call.
About the 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 1 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 United States, more than 80,000 patients treated
with oral Factor Xa inhibitors were admitted to the hospital due to
bleeding during 2015. Including patients taking the injectable
Factor Xa inhibitor enoxaparin, it is estimated that more than
100,000 U.S. patients could benefit from an antidote annually.
Currently, there is no FDA-approved antidote for Factor Xa
inhibitors for these patients.
About AndexXa AndexXa, 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. AndexXa 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.
Portola’s BLA for AndexXa was based on data from two Phase 3
ANNEXA™ studies that evaluated the safety and efficacy of AndexXa
in reversing the anticoagulant activity of the Factor Xa inhibitors
rivaroxaban and apixaban in older healthy volunteers. Results of
those studies were published online by The New England Journal of
Medicine in November 2015.
The BLA also included limited adjudicated efficacy and safety
data from initial patients enrolled in the ongoing ANNEXA-4 study.
Portola is currently evaluating AndexXa in this global, Phase 3b/4
single-arm, open-label confirmatory study in patients receiving
apixaban, rivaroxaban, edoxaban or enoxaparin who present with an
acute major bleed.
The FDA granted AndexXa Orphan Drug designation, for which
Portola would expect to receive seven years of marketing
exclusivity if the drug is approved. This designation applies to
drugs and biologics intended for the treatment, diagnosis or
prevention of rare diseases/disorders that affect fewer than
200,000 people in the United States.
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; AndexXa™ (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 AndexXa. Risks that contribute to the uncertain nature
of the forward-looking statements include the risk that we may be
unable to satisfy the FDA’s requirements for such approval. 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.
Investor Contact:
Ana Kapor
Portola Pharmaceuticals
ir@portola.com
Media Contact:
Julie Normart
W2O Group
jnormart@w2ogroup.com
Portola Pharmaceuticals (NASDAQ:PTLA)
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