SAN FRANCISCO, Dec. 1, 2014 /PRNewswire/ -- Nektar
Therapeutics (NASDAQ:NKTR) reported that partner Baxter
International Inc. today announced that the company has submitted a
biologics license application (BLA) to the United States Food and
Drug Administration (FDA) for the approval of BAX 855, an
investigational, extended half-life recombinant factor VIII
(rFVIII) treatment for hemophilia A based on ADVATE [Antihemophilic
Factor (Recombinant)].
"The evidence from our late-stage controlled trial showcases
that BAX 855 could become an important new treatment option for
people with hemophilia A, expanding on more than a decade of
experience with ADVATE," said John
Orloff, M.D., vice president and global head of research and
development for Baxter BioScience. "BAX 855 represents our
continued commitment to developing valuable new options for the
hemophilia community that address the needs of patients around the
world."
The submission is based on positive results from a prospective,
global, multi-center, open-label, two-arm Phase 3 study of 137
previously treated patients (PTP). The results demonstrated that
BAX 855 met its primary endpoint in the control and prevention of
bleeding episodes and routine prophylaxis for patients who were 12
years or older. Patients in a twice-weekly prophylaxis arm
experienced a 95 percent reduction in median ABR as compared to
those in the on-demand arm (1.9 vs. 41.5, respectively). BAX 855
was also effective in treating bleeding episodes, 96 percent of
which were controlled with one or two infusions. No patients
developed inhibitors to BAX 855 and no treatment-related serious
adverse events, including hypersensitivity, were reported. The most
common (three patients) product-related adverse reaction was
headache.
Baxter expects to present
additional data from the BAX 855 pivotal trial in the coming
months. The continuation study remains ongoing for patients who
completed the pivotal trial and the Phase 3 study among previously
treated patients under the age of 12 with severe hemophilia A is
progressing. Once the pediatric study is complete, Baxter expects to file for marketing
authorization with the European Medicines Agency in 2016. Upon
approval, BAX 855 is expected to be produced at the company's
Singapore manufacturing facility,
one of several sites currently approved for ADVATE
production.
BAX 855 is based on ADVATE, a full-length FVIII molecule with
more than 10 years of real-world experience. Through a
collaboration with Nektar Therapeutics (NASDAQ: NKTR), BAX 855
leverages proprietary PEGylation technology designed to extend the
duration of activity of proteins in the body. This proprietary
technology has been used for over 10 years in a number of approved
medicines that treat chronic or serious conditions.
About ADVATE
ADVATE is a recombinant antihemophilic factor indicated for use
in children and adults with hemophilia A (congenital factor VIII
deficiency or classic hemophilia) for:
- Control and prevention of bleeding episodes.
- Perioperative management.
- Routine prophylaxis to prevent or reduce the frequency of
bleeding episodes.
ADVATE is not indicated for the treatment of von Willebrand
disease.
ADVATE has a demonstrated efficacy and safety profile. ADVATE is
a full-length (derived from the complete FVIII gene) recombinant
FVIII product that is processed without any blood-based additives.
Because no blood-derived components are added at any stage of the
manufacturing process, the potential risk of transmitting pathogens
that may be carried in blood-based additives is eliminated. There
have been no confirmed reports of transmission of HIV, HBV or HCV
with rFVIII treatments.
ADVATE is the world's most prescribed FVIII treatment. It is
currently approved in 64 countries worldwide, including
the United States, Canada, 28 countries in the European Union,
Algeria, Argentina, Australia, Brazil, Chile, China,
Colombia, Ecuador, Hong
Kong, Iceland, Iraq, Israel,
Japan, Kuwait, Macau, Malaysia, Mexico, Morocco, New
Zealand, Norway,
Panama, Puerto Rico, Russia, Saudi
Arabia, Serbia, Singapore,
South Korea, Suriname,
Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.
Detailed Important Risk Information for ADVATE
[Antihemophilic Factor (Recombinant)]
CONTRAINDICATIONS
ADVATE is contraindicated in patients who have life-threatening
hypersensitivity reactions, including anaphylaxis, to mouse or
hamster protein or other constituents of the product.
WARNINGS & PRECAUTIONS
Hypersensitivity Reactions
Allergic-type hypersensitivity reactions, including anaphylaxis,
have been reported with ADVATE. Symptoms include dizziness,
paresthesia, rash, flushing, facial swelling, urticaria, dyspnea,
and pruritus.
Discontinue ADVATE if hypersensitivity symptoms occur and
administer appropriate emergency treatment.
Neutralizing Antibodies
Neutralizing antibodies (inhibitors) have been reported
following administration of ADVATE predominantly in previously
untreated patients (PUPs) and previously minimally treated patients
(MTPs). Monitor all patients for the development of factor VIII
inhibitors by appropriate clinical observation and laboratory
testing. If expected plasma factor VIII activity levels are not
attained, or if bleeding is not controlled with an expected dose,
perform an assay that measures factor VIII inhibitor
concentration.
ADVERSE REACTIONS
The serious adverse reactions seen with ADVATE are
hypersensitivity reactions and the development of high-titer
inhibitors necessitating alternative treatments to factor VIII.The
most common adverse reactions observed in clinical trials
(frequency ≥10% of subjects) were pyrexia, headache, cough,
nasopharyngitis, vomiting, arthralgia, and limb injury. Please
see full prescribing information for ADVATE at:
http://www.baxter.com/downloads/healthcare_professionals/products/ADVATE_PI.pdf
About Baxter in
Hemophilia
Baxter has more than 60 years
experience in hemophilia and has introduced a number of therapeutic
firsts for hemophilia patients. Baxter has the broadest portfolio of
hemophilia treatments in the industry and is able to meet
individual therapy choices, providing a range of options at each
treatment stage. The company's work focuses on optimizing
hemophilia care and improving the lives of people worldwide living
with bleeding disorders.
About Baxter International Inc.
Baxter International Inc., through its subsidiaries, develops,
manufactures and markets products that save and sustain the lives
of people with hemophilia, immune disorders, cancer, infectious
diseases, kidney disease, trauma and other chronic and acute
medical conditions. As a global, diversified healthcare company,
Baxter applies a unique
combination of expertise in medical devices, pharmaceuticals and
biotechnology to create products that advance patient care
worldwide.
This release includes forward-looking statements concerning
BAX 855, including expectations with regard to clinical studies and
related data, regulatory filings, future manufacturing plans and
its potential impact to patients. The statements are based on
assumptions about many important factors, including the following,
which could cause actual results to differ materially from those in
the forward-looking statements: satisfaction of regulatory and
other requirements; actions of regulatory bodies and other
governmental authorities; additional clinical results; changes in
laws and regulations; product quality or supply or patient safety
issues; and other risks identified in Baxter's most recent filing on Form 10-K and
other SEC filings, all of which are available on Baxter's website. Baxter does not undertake to update its
forward-looking statements.
Media Contact
Brian Kyhos
(224) 948-5353, media@baxter.com
Investor Contact
Mary Kay
Ladone, (224) 948-3371
Clare Trachtman, (224) 948-3085
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SOURCE Baxter International Inc.