DEERFIELD, Ill., April 16, 2015 /PRNewswire/ -- Nektar
Therapeutics (NASDAQ:NKTR) reported today that partner Baxter
International Inc. announced that the company has submitted a new
drug application (NDA) to Japan's
Ministry of Health, Labour and Welfare for the approval of BAX 855,
an investigational, extended half-life recombinant factor VIII
(rFVIII) treatment based on ADVATE [Antihemophilic Factor
(Recombinant)] for patients over 12 years of age with hemophilia
A.
"We continue to advance BAX 855 as a potential new treatment for
hemophilia A patients around the world who are seeking options that
support their individual needs," said John
Orloff, M.D., vice president and global head of research and
development for Baxter BioScience. "With more than a decade of
experience with ADVATE and an extensive global presence, our BAX
855 program represents our continued commitment to supporting the
hemophilia community, particularly this week as we celebrate World
Hemophilia Day with our colleagues around the globe."
The submission follows the filing to the United States Food and
Drug Administration (FDA) in late 2014 and is based on positive
results from a prospective, global, Phase 3 study of 137 previously
treated patients (PTP). The results, presented during the European
Association for Haemophilia and Allied Disorders (EAHAD) meeting in
February 2015, 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 annual bleed rate (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 product-related adverse reaction was headache (3
patients).
Baxter continues to advance a continuation study for patients
who completed the pivotal trial and a Phase 3 study among
previously treated patients under the age of 12 with severe
hemophilia A. Once the pediatric study has been completed, the
company expects to file for marketing authorization with the
European Medicines Agency in 2016 and intends to seek post-approval
label expansion in the U.S. for previously-untreated pediatric
patients.
BAX 855 is based on ADVATE, a full-length FVIII molecule with
more than 12 years of real-world experience. Through a
collaboration with Nektar Therapeutics, BAX 855 leverages
proprietary pegylation technology designed to extend the duration
of activity of the protein 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, 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, manufacturing or supply issues, 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
Kellie Hotz
(224) 948-5353, media@baxter.com
Investor Contacts
Mary Kay Ladone, (224) 948-3371
Clare Trachtman, (224) 948-3085
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SOURCE Nektar Therapeutics