Portola Pharmaceuticals Inc.® (Nasdaq:PTLA) today announced the
U.S. Food and Drug Administration (FDA) has approved Bevyxxa
(betrixaban), the first and only anticoagulant for hospital and
extended duration prophylaxis (35 to 42 days) of venous
thromboembolism (VTE) in adult patients hospitalized for an acute
medical illness who are at risk for thromboembolic complications
due to moderate or severe restricted mobility and other risk
factors for VTE.
Bevyxxa, an oral, once-daily Factor Xa inhibitor, was granted a
Fast Track designation and approved by the FDA under Priority
Review, which is a status given to drugs that may offer significant
improvements in treatment or provide a treatment where no adequate
therapy exists. Bevyxxa has been approved based on data from
Portola's pivotal Phase 3 APEX Study, which enrolled 7,513 patients
at more than 450 clinical sites worldwide.
"Bevyxxa represents a major advance for the field of thrombosis.
It is the first therapy to demonstrate a reduction in the incidence
of VTE in these high-risk patients without a significant increase
in major bleeding,” said C. Michael Gibson, M.D., APEX Executive
Committee Member and Steering Committee Chairman, professor,
Harvard Medical School and chairman of the PERFUSE Study Group.
“With this approval, we are finally able to help protect these
patients from this often fatal, yet preventable condition.”
“Our goal as a company is to bring to market important medicines
for the benefit of patients,” said Bill Lis, Chief Executive
Officer of Portola. “Today’s approval is the ultimate milestone for
Portola. We are grateful to the patients who participated in our
trials, the FDA, our academic collaborators and investigators, and,
importantly, our dedicated employees who have worked tirelessly to
achieve this goal.”
Acutely ill medical patients are those hospitalized for serious
medical conditions, including heart failure, stroke, infection and
pulmonary disease. Because of their underlying disorder and
immobilization, they are at increased risk of developing deep vein
thrombosis (DVT) and pulmonary embolism (PE) blood clots.
In the G7 countries, an estimated 24 million acutely ill medical
patients are hospitalized each year and are at risk of VTE, either
while in the hospital or following discharge. More than one million
VTE events and 150,000 VTE-related deaths occur annually in acutely
ill medical 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 patients are discharged from
the hospital. No other anticoagulant, including enoxaparin or any
of the marketed oral Factor Xa inhibitors, is approved for
in-hospital and extended-duration VTE prophylaxis in acutely ill
medical patients.
The APEX study evaluated oral betrixaban for 35 to 42 days
compared with injectable enoxaparin for 6 to 14 days followed by
placebo in assessing the prevention of VTE in high-risk acutely ill
medical patients. As detailed in the prescribing information,
Bevyxxa efficacy was measured in the modified Intent-to-Treat
(mITT) analysis, which includes 7,441 patients assessed by a
composite outcome score comprising either the occurrence of
asymptomatic proximal DVT or symptomatic DVT, non-fatal PE or
VTE-related death. Bevyxxa reduced the incidence of DVT and PE
blood clots compared with those taking enoxaparin plus placebo (4.4
percent vs. 6.0 percent; relative risk 0.75, 95 percent CI: 0.61,
0.91) with no significant increase in major bleeding (0.67 percent
vs. 0.57 percent). The most frequent reason for treatment
discontinuation was bleeding, with an incidence rate for all
bleeding episodes of 2.4 percent and 1.2 percent for betrixaban and
enoxaparin, respectively.
Results from the APEX Study have been peer-reviewed and
published in The New England Journal of Medicine, Circulation and
the American Heart Journal.1
“For the first time, physicians will have a therapy to help
reduce VTE in acutely ill medical patients during their transition
from hospital to home, which may ultimately help reduce morbidity,”
said Alexander (Ander) T. Cohen, M.B.B.S., M.Sc., M.D., FRACP, APEX
Co-Principal Investigator and Co-Chairman of the APEX Executive
Committee and Consultant Physician at Guy’s and St Thomas’ NHS
Foundation.
The timeline on which Portola expects to launch Bevyxxa is
between August and November 2017. During this period, Portola will
complete salesforce hiring and training, drug manufacturing
validation and inventory buildup. For more information regarding
the availability of Bevyxxa, please go to www.bevyxxa.com.
In the EU, the European Medicines Agency’s Committee for
Human Medicinal Products (CHMP) is reviewing the Marketing
Authorization Application for betrixaban under its standard review
period.
CONFERENCE CALL The Portola management team
will host a conference call and webcast today, June 23, 2017, to
provide more information about Bevyxxa. The live call can be
accessed by phone by calling (844) 452-6828 (domestic) or (765)
507-2588 (international) and specifying conference call ID
45842131. 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.
BEVYXXA INDICATION AND USEBevyxxa (betrixaban)
is indicated for the prophylaxis of VTE in adult patients
hospitalized for an acute medical illness who are at risk for
thromboembolic complications due to moderate or severe restricted
mobility and other risk factors for VTE.
The recommended dose of Bevyxxa is an initial single dose of 160
mg starting on day 1, followed by 80 mg once daily taken for 35 to
42 days at the same time each day with food.
Limitations of UseThe safety and effectiveness
of Bevyxxa have not been established in patients with prosthetic
heart valves because this population has not been studied.
IMPORTANT SAFETY INFORMATION FOR BEVYXXA
Warning: Spinal / Epidural
HematomaEpidural or spinal hematomas may occur in
patients treated with betrixaban who are receiving neuraxial
anesthesia or undergoing spinal puncture. The risk of these events
may be increased by the use of in-dwelling epidural catheters or
the concomitant use of medical products affecting hemostasis. These
hematomas may result in long-term or permanent paralysis. Consider
these risks when scheduling patients for spinal
procedures.
CONTRAINDICATIONSActive pathological bleeding;
severe hypersensitivity reaction to Bevyxxa.
WARNINGS AND PRECAUTIONS
Risk of BleedingBevyxxa increases the risk of
bleeding and can cause serious and potentially fatal bleeding;
concomitant use of drugs affecting hemostasis increases the risk of
bleeding. These include aspirin and other antiplatelet agents,
other anticoagulants, heparin, thrombolytic agents, selective
serotonin reuptake inhibitors, serotonin norepinephrine reuptake
inhibitors, and nonsteroidal anti-inflammatory drugs (NSAIDs).
Advise patients of signs and symptoms of blood loss and to
report them immediately or go to an emergency room. Promptly
evaluate any signs or symptoms of blood loss and consider the need
for blood replacement. Discontinue Bevyxxa in patients with active
pathological bleeding. There is no established way to reverse the
anticoagulant effect of betrixaban, which can be expected to
persist for at least 72 hours after the last dose.
Spinal/Epidural Anesthesia or PunctureWhen
neuraxial anesthesia (spinal/epidural anesthesia) or
spinal/epidural puncture is employed, patients treated with
antithrombotic agents for prevention of thromboembolic
complications are at risk of developing an epidural or spinal
hematoma which can result in long-term or permanent paralysis. An
epidural catheter should not be removed earlier than 72 hours
after the last administration of Bevyxxa. The next Bevyxxa dose is
not to be administered earlier than 5 hours after the removal
of the catheter. If traumatic puncture occurs, delay the
administration of Bevyxxa for 7 hours. Monitor patients
frequently for signs and symptoms of neurological impairment
(e.g., numbness or weakness of the legs, bowel or bladder
dysfunction). If neurological compromise is noted, urgent
diagnosis and treatment is necessary.
Use in Patients with Severe Renal
ImpairmentPatients with severe renal impairment (CrCl ≥ 15
to < 30 mL/min computed by Cockcroft-Gault) taking Bevyxxa may
have an increased risk of bleeding events. Reduce dose of
Bevyxxa, monitor patients closely, and promptly evaluate any signs
or symptoms of blood loss in these patients.
Use in Patients on Concomitant P-glycoprotein (P-gp)
InhibitorsPatients on concomitant P-gp inhibitors with
Bevyxxa may have an increased risk of bleeding. Reduce dose of
Bevyxxa, monitor patients closely, and promptly evaluate any signs
or symptoms of blood loss in these patients. Avoid use of Bevyxxa
in patients with severe renal impairment receiving concomitant P‑gp
inhibitors.
ADVERSE REACTIONSThe most common adverse
reactions with Bevyxxa were related to bleeding (> 5
percent).
USE IN SPECIFIC POPULATIONSHepatic
ImpairmentBevyxxa has not been evaluated in patients with
hepatic impairment, because these patients may have intrinsic
coagulation abnormalities. Bevyxxa is not recommended in patients
with hepatic impairment.
For additional information and full Prescribing Information for
Bevyxxa, please visit http://www.bevyxxa.com
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’s first
commercial product, Bevyxxa (betrixaban), an oral, once-daily
Factor Xa inhibitor anticoagulant, is approved in the United
States. Portola is advancing the clinical development of two other
compounds, including 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 results that may be achieved after
treatment with Bevyxxa and the timing of the availability of
Bevyxxa to physicians and their patients in the United States.
Risks that contribute to the uncertain nature of the
forward-looking statements include our manufacturers’ ability to
manufacture Bevyxxa on a commercial scale or scale to increased
production and our overall ability to effectively commercialize
Bevyxxa. 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 May 8, 2017. 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.
1 Cohen, Alexander T., Robert A. Harrington, Samuel Z.
Goldhaber, Russell D. Hull, Brian L. Wiens, Alex Gold, Adrian F.
Hernandez, and C. Michael Gibson. "Extended Thromboprophylaxis with
Betrixaban in Acutely Ill Medical Patients." New England Journal of
Medicine 375.6 (2016)
Investor Contact:
Ana Kapor
Portola Pharmaceuticals
ir@portola.com
Media Contact:
Julie Normart
W2O Group
jnormart@purecommunications.com
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