Portola Pharmaceuticals Announces FDA Accepts New Drug Application for Priority Review and EMA Validates Marketing Authorizat...
December 23 2016 - 2:12PM
Portola Pharmaceuticals, Inc.® (Nasdaq:PTLA) today announced that
the U.S. Food and Drug Administration (FDA) accepted Portola’s New
Drug Application (NDA) granting priority review for betrixaban, an
oral, once-daily Factor Xa inhibitor anticoagulant, for
extended-duration prophylaxis of venous thromboembolism (VTE) in
acute medically ill patients with risk factors for VTE. A priority
review shortens the FDA review timeline to six months from the
standard review period of 10 months. The application for
betrixaban, an FDA-designated Fast Track investigational drug, was
deemed sufficiently complete to permit a substantive review and has
been given a Prescription Drug User Fee Act (PDUFA) action date of
June 24, 2017.
Additionally, Portola announced that the European Medicines
Agency (EMA) has validated its Marketing Authorization Application
(MAA) for betrixaban for extended-duration prophylaxis of VTE in
adults with acute medical illness and risk factors for VTE. The
EMA’s Committee for Medicinal Products for Human Use (CHMP) is
reviewing the application under a standard 210-day review
period.
“With the filing of the betrixaban NDA and the MAA validation,
we now look forward to working with the FDA and EMA to bring this
drug to market,” said Bill Lis, chief executive officer of Portola.
“Betrixaban has the potential to be the first anticoagulant
approved for in-hospital and extended-duration VTE prophylaxis in
high-risk acute medically ill patients.”
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 International Society on Thrombosis and Haemostasis (ISTH)
Scientific and Standardization Committee (SSC) Meeting in May 2016
and published online in The New England Journal of Medicine in May
2016.i Results from three sub-studies of the APEX Study,
including a retrospective sub-study that assessed the potential of
extended-duration thromboprophylaxis with betrixaban to reduce the
risk of stroke in hospitalized acute medically ill patients, were
presented at the American Heart Association (AHA) Scientific
Sessions in November 2016 and published in Circulation.ii
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.
About Betrixaban
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.
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 Statements
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 the United States and foreign countries; 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, which was filed on November 7, 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 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.
Investor Contact:
Ana Kapor
Portola Pharmaceuticals
ir@portola.com
Media Contact:
Julie Normart
W2O Group
jnormart@w2ogroup.com
415.946.1087
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