SOUTH SAN FRANCISCO, Calif.,
Aug. 5, 2019 /PRNewswire/
-- Portola Pharmaceuticals, Inc.® (Nasdaq: PTLA)
today announced that the U.S. Centers for Medicare and Medicaid
Services (CMS) has increased the maximum reimbursement amount for
the New Technology Add-on Payment (NTAP) granted in October 2018 to Andexxa® [coagulation
factor Xa (recombinant), inactivated-zhzo] from 50 percent to 65
percent.
The final rule concerning the Hospital Inpatient Prospective
Payment System and CMS Fiscal Year 2020 was pre-published online
Friday, August 2, 2019 (pages
420-422)
(https://s3.amazonaws.com/public-inspection.federalregister.gov/2019-16762.pdf),
and will be formally published in the Federal Register on
Friday, August 16, 2019.
"Today's CMS decision supports the breakthrough innovation of
Andexxa and the clinical value of rapidly reversing the
anticoagulant effects of the Factor Xa inhibitors rivaroxaban and
apixaban in the event of life-threatening or uncontrolled
bleeding," said Scott Garland,
Portola's president and chief
executive officer. "We believe CMS' decision to increase the
reimbursement rate for Andexxa will both expand Medicare
beneficiary access to Andexxa and support the hospitals that are
incorporating this important therapy into their treatment
protocols."
Introduced in 2001, the CMS NTAP program was created by Congress
to support timely access to innovative therapies used to treat
Medicare beneficiaries in the hospital inpatient setting. For a new
technology to qualify for an add-on payment, it must meet the NTAP
definition of "new," demonstrate a substantial clinical improvement
and meet specific cost thresholds.
Beginning October 1, 2019, the
maximum NTAP reimbursement for a qualifying case involving the use
of Andexxa is up to $18,281.25, or 65
percent of the wholesale acquisition cost of the standard dose.
NTAP is expected to remain in effect for a period of two to three
years until the cost of Andexxa is included in the recalibration of
the diagnosis-related group (DRG) payment rates.
About Factor Xa Inhibitor-Related Bleeding
The use of
Factor Xa inhibitors is growing rapidly because of their efficacy
and safety profile compared to enoxaparin and warfarin in
preventing and treating thromboembolic conditions such as stroke,
pulmonary embolism and venous thromboembolism (VTE). This growth
has come with a related increase in the incidence of hospital
admissions and deaths related to bleeding, the major complication
of anticoagulation. In 2017, there were approximately 150,000
hospital admissions attributable to Factor Xa inhibitor-related
bleeding and approximately 2,100 bleeding-related deaths per month
in the U.S.
About Andexxa
Andexxa is a recombinant protein
specifically designed to bind to Factor Xa inhibitors and rapidly
reverse their anticoagulant effect. Andexxa was approved by the FDA
in May 2018 as the first and only
antidote indicated for patients treated with rivaroxaban and
apixaban, when reversal of anticoagulation is needed due to
life-threatening or uncontrolled bleeding. Andexxa received both
U.S. Orphan Drug and FDA Breakthrough Therapy designations and was
approved under the FDA's Accelerated Approval pathway based on the
change from baseline in anti-Factor Xa activity in healthy
volunteers.
For additional Important Safety Information and Andexxa's full
Prescribing Information, please visit http://www.Andexxa.com.
IMPORTANT INFORMATION FOR ANDEXXA [coagulation factor Xa
(recombinant), inactivated-zhzo]
BOXED WARNING: THROMBOEMBOLIC RISKS, ISCHEMIC RISKS, CARDIAC
ARREST AND SUDDEN DEATHS
See full prescribing information for
complete boxed warning
Treatment with Andexxa has been associated with serious and
life‑threatening adverse events, including:
- Arterial and venous thromboembolic events
- Ischemic events, including myocardial infarction and
ischemic stroke
- Cardiac arrest
- Sudden deaths
Monitor for thromboembolic events and initiate
anticoagulation when medically appropriate. Monitor for symptoms
and signs that precede cardiac arrest and provide treatment as
needed.
Indication
Andexxa [coagulation factor Xa
(recombinant), inactivated-zhzo] is indicated for patients
treated with rivaroxaban and apixaban, when reversal of
anticoagulation is needed due to life-threatening or uncontrolled
bleeding.
This indication is approved under accelerated approval based on
the change from baseline in anti-Factor Xa (FXa) activity in
healthy volunteers. An improvement in hemostasis has not been
established. Continued approval for this indication may be
contingent upon the results of studies to demonstrate an
improvement in hemostasis in patients.
Andexxa has not been shown to be effective for, and is not
indicated for, the treatment of bleeding related to any FXa
inhibitors other than apixaban and rivaroxaban.
SELECT IMPORTANT SAFETY INFORMATION
Thromboembolic Risk
Arterial and venous thromboembolic
events, ischemic events, sudden deaths, or events where a
thrombotic event could not be ruled out were observed within 30
days post- Andexxa administration in 33 of the 185 patients
(17.8%) evaluable for safety in the ongoing ANNEXA-4 study. The
median time to these events was six days. Of the 86 patients who
were re-anticoagulated prior to a thrombotic event, 11 (12.7%)
patients experienced a thromboembolic event, ischemic event,
cardiac event or death.
Monitor patients treated with Andexxa for signs and
symptoms of arterial and venous thromboembolic events, ischemic
events, and cardiac arrest. To reduce thromboembolic risk, resume
anticoagulant therapy as soon as medically appropriate following
treatment with Andexxa. No thromboembolic events were observed in
223 healthy volunteers who received Factor Xa inhibitors and were
treated with Andexxa.
The safety of Andexxa has not been evaluated in patients
who experienced thromboembolic events or disseminated intravascular
coagulation within two weeks prior to the life-threatening bleeding
event requiring treatment with Andexxa. Safety of Andexxa also
has not been evaluated in patients who received prothrombin complex
concentrates, recombinant Factor VIIa, or whole blood products
within seven days prior to the bleeding event.
Re-elevation or Incomplete Reversal of Anti-FXa
Activity
The time course of anti-FXa activity following
Andexxa administration was consistent among the healthy
volunteer studies and the ANNEXA-4 study in bleeding patients.
Compared to baseline, there was a rapid and substantial decrease in
anti-FXa activity corresponding to the Andexxa bolus. This
decrease was sustained through the end of the
Andexxa continuous infusion. Following the infusion, there was
an increase in anti-FXa activity, which peaked four hours after
infusion in ANNEXA-4 subjects. After this peak, the anti-FXa
activity decreased at a rate similar to the clearance of the FXa
inhibitors.
Thirty-eight patients who were anticoagulated with apixaban had
baseline levels of anti-FXa activity > 150 ng/mL. Nineteen of
these 38 (50%) patients experienced a > 93% decrease from
baseline anti-FXa activity after administration of Andexxa. Eleven
patients who were anticoagulated with rivaroxaban had baseline
anti-FXa activity levels > 300 ng/mL. Five of the 11 patients
experienced a > 90% decrease from baseline anti-FXa activity
after administration of Andexxa.
Adverse Reactions
The most common adverse reactions (≥
5%) in patients receiving Andexxa were urinary tract infections and
pneumonia.
The most common adverse reactions (≥ 3%) in healthy volunteers
treated with Andexxa were infusion-related reactions.
Immunogenicity
As with all therapeutic proteins, there
is potential for immunogenicity. Low titers of anti-Andexxa
antibodies were observed in 26/145 healthy subjects (17%); 6%
(9/145) were first observed at Day 30 with 20 subjects (14%) still
having titers at the last time point (days 44 to 48). To date, the
pattern of antibody response in patients in the ANNEXA-4 study has
been similar to that observed in healthy volunteers with 6% of the
patients having antibodies against Andexxa (6/98 patients). None of
these anti-Andexxa antibodies were neutralizing. No antibodies
cross-reacting with FX or FXa were detected in healthy subjects
(0/145) or in bleeding patients to date (0/98).
About Portola Pharmaceuticals, Inc.
Portola
Pharmaceuticals is a global, 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 conditions.
The Company's first two commercialized products are
Andexxa® [coagulation factor Xa (recombinant),
inactivated-zhzo], marketed in Europe as Ondexxya® (andexanet
alfa), and Bevyxxa® (betrixaban). The company also
is advancing cerdulatinib, a SYK/JAK inhibitor being developed for
the treatment of hematologic cancers. Founded in 2003 in South
San Francisco, California,
Portola has operations in
the United States and
Europe.
Forward-Looking Statements
This announcement contains
forward-looking statements, including statements relating to
Portola Pharmaceuticals' expectations regarding the potential
commercial and medical impact of receiving the NTAP for Andexxa.
These statements are subject to significant risks and
uncertainties, and actual results could differ materially from
those projected. Portola Pharmaceuticals cautions investors not to
place undue reliance on the forward-looking statements contained in
this release. These risks and uncertainties include, without
limitation, risks and uncertainties that physicians may not see the
benefits of utilizing Andexxa for the indications which it is
approved; the ability of Portola
to continue to manufacture Andexxa and to expand approved
manufacturing facilities; the possibility of unfavorable results
from additional clinical trials involving Andexxa; the amount of
time that the NTAP is available; the risk that the EMA may not
approve Andexxa in the currently anticipated timelines or at all,
and that any marketing approvals may have significant limitations
on its use; the risk that Portola
may not obtain additional regulatory approvals necessary to expand
approved indications for Andexxa; and other general business risks
which could have a material adverse impact on Portola's business, including risks associated
with the launch of Portola's first
product Bevyxxa®; regulatory actions or delays or
government regulation generally; Portola's ability to obtain or maintain
proprietary intellectual property protection; the particular
prescribing preferences of physicians and patients; global trends
toward health care cost containment, including government, payor
and general public pricing and reimbursement pressures; general
economic and industry conditions, including the effects of the
persistently weak economic and financial environment in many
countries; safety, quality or manufacturing issues. Risks and
uncertainties relating to Portola Pharmaceuticals and its
business can be found in the "Risk Factors" section of Portola
Pharmaceuticals' Annual Report on Form 10-K for 2017, which was
filed with the SEC on March 1, 2018, as updated by
subsequent periodic reports filed by Portola with the SEC, including Quarterly
Reports on Form 10-Q and Current Reports on Form 8-K which are
deemed "filed" with the SEC. Portola Pharmaceuticals
undertakes no duty or obligation to update any forward-looking
statements contained in this release as a result of new
information, future events or changes in Portola Pharmaceuticals'
expectations.
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SOURCE Portola Pharmaceuticals, Inc.®