BANNOCKBURN, Ill., Nov. 16, 2015 /PRNewswire/ -- Baxalta
Incorporated (NYSE: BXLT), a global biopharmaceutical leader
dedicated to delivering transformative therapies to patients with
orphan diseases and underserved conditions, announced today that
the U.S. Food and Drug Administration (FDA) has approved ADYNOVATE
[Antihemophilic Factor (Recombinant), PEGylated], an extended
circulating half-life recombinant Factor VIII (rFVIII) treatment
for hemophilia A. ADYNOVATE is built on the full-length ADVATE
[Antihemophilic Factor (Recombinant)] molecule, a leading treatment
for hemophilia A with more than 12 years of real-world patient
experience.
"People with hemophilia want to minimize their bleeds and not
let their hemophilia dictate their lives; having options empowers
patients to make the best decisions for their personal care," said
Val Bias, chief executive officer,
National Hemophilia Foundation. "We applaud Baxalta for continuing
to innovate for the care of hemophilia."
"ADYNOVATE represents Baxalta's commitment to meeting the needs
of hemophilia A patients who want bleed reduction with a dosing
schedule that better fits their personal needs," said Ludwig Hantson, chief executive officer and
president, Baxalta. "As the first new product approved under the
Baxalta name, ADYNOVATE represents a major milestone in achieving
our goal of 20 product launches by 2020 and continuing to expand
our world-leading hemophilia treatment portfolio."
In the pivotal phase 3 clinical trial, which served as the
foundation for the approval, ADYNOVATE demonstrated efficacy in
treating hemophilia patients through routine prophylaxis as well as
for on-demand bleeding episodes. Patients, 12 to 65 years of age,
in the prospective, global, multi-center, open label,
non-randomized study were assigned to either twice weekly
prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60
IU/kg, n=17) with ADYNOVATE. The study found that
previously-treated patients in a twice-weekly prophylaxis arm had
95 percent fewer annual bleeds compared to those treated on-demand
[median annual bleed rate (ABR) 1.9 vs. 41.5, respectively]. During
the study, 38 percent (n=120) of prophylaxis-treated patients
experienced zero bleeds. Moreover, 57 percent of patients
experienced zero joint bleeds based on six months of prophylaxis
with ADYNOVATE.2
Nearly all (98 percent) of patients on prophylaxis with
ADYNOVATE did not have a dose adjustment in the study. Nearly all
(96 percent) bleeding episodes (n=591) were controlled with one or
two infusions of ADYNOVATE. No patients developed inhibitors to the
treatment; the most common adverse reactions (≥1 percent of
subjects) were headache and nausea.2
ADYNOVATE will be available in the
United States in the coming weeks. Baxalta continues to
invest in ADYNOVATE to expand the product's value for more patients
worldwide. Currently, studies are ongoing in previously treated
patients (PTPs) with severe hemophilia A undergoing surgery and in
pediatric PTPs under the age of 12 with severe hemophilia A.
Additionally, Baxalta will initiate a study in previously-untreated
patients (PUPs) with severe hemophilia A. Baxalta has filed for
regulatory approval of the treatment in Japan and following completion of the
pediatric study, expects to file for marketing authorization in
Europe.
Hemophilia A is a challenging chronic disease; treatment
regimens require regular infusions to reduce the risk of bleeding.
Working closely with their health professionals, many patients
continue to seek treatment options that can be better personalized
to fit their needs, providing both effective bleed protection and
simplified dosing schedules. Today, the disease affects
approximately 16,000 people in the United
States and more than 400,000 people worldwide. With an
estimated 75 percent of people with hemophilia undiagnosed and
untreated or undertreated globally,3 Baxalta continues
to innovate in order to address some of the greatest challenges
associated with hematologic disorders, including hemophilia.
Through a collaboration with Nektar Therapeutics (NASDAQ: NKTR),
ADYNOVATE leverages proprietary pegylation technology designed to
prolong the amount of FVIII available for use in the body. The
technology was selected because it maintains the integrity of the
parent molecule (ADVATE) and reduces the speed at which the body
clears ADYNOVATE, resulting in increased circulating half-life.
This proprietary technology has been used for more than 15 years in
a number of approved medicines that treat chronic or serious
conditions. In addition to the patents relating to pegylated FVIII,
Baxalta has an exclusive license in the field of hemophilia to
certain other patents relating to pegylated FVIII proteins. These
patents protect Baxalta's pipeline of extended circulating
half-life FVIII products, including ADYNOVATE.
About ADYNOVATE
ADYNOVATE, Antihemophilic Factor (Recombinant), PEGylated, is a
human antihemophilic factor indicated in adolescent and adult
patients (12 years and older) with hemophilia A (congenital factor
VIII deficiency) for:
- On-demand treatment and control of bleeding episodes
- Routine prophylaxis to reduce the frequency of bleeding
episodes
ADYNOVATE is not indicated for the treatment of von Willebrand
disease.
DETAILED IMPORTANT RISK
INFORMATION
CONTRAINDICATIONS
ADYNOVATE is contraindicated in patients who have had prior
anaphylactic reaction to ADYNOVATE, to the parent molecule
(ADVATE), mouse or hamster protein, or excipients of ADYNOVATE
(e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate
80).
WARNINGS & PRECAUTIONS
Hypersensitivity Reactions
Hypersensitivity reactions are possible with ADYNOVATE.
Allergic-type hypersensitivity reactions, including anaphylaxis,
have been reported with other recombinant antihemophilic factor
VIII products, including the parent molecule, ADVATE. Early signs
of hypersensitivity reactions that can progress to anaphylaxis may
include angioedema, chest tightness, dyspnea, wheezing, urticaria,
and pruritus. Immediately discontinue administration and initiate
appropriate treatment if hypersensitivity reactions occur.
Neutralizing Antibodies
Formation of neutralizing antibodies (inhibitors) to factor VIII
can occur following administration of ADYNOVATE. Monitor patients
regularly for the development of factor VIII inhibitors by
appropriate clinical observations and laboratory tests. Perform an
assay that measures factor VIII inhibitor concentration if the
plasma factor VIII level fails to increase as expected, or if
bleeding is not controlled with expected dose.
ADVERSE REACTIONS
Common adverse reactions (≥1% of subjects) reported in the
clinical studies were headache and nausea.
For Full Prescribing Information, visit
http://baxalta.com/assets/documents/ADYNOVATE_PI.pdf.
About ADVATE
ADVATE [Antihemophilic Factor (Recombinant)] is a recombinant
antihemophilic factor indicated for use in children and adults with
hemophilia A (congenital factor VIII deficiency) 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 for the
treatment of hemophilia A. 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 virtually
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,
pruritus, and vomiting.
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
Serious adverse reactions seen with ADVATE are hypersensitivity
reactions, including anaphylaxis, and the development of high-titer
inhibitors necessitating alternative treatments to factor VIII.
The most common adverse reactions observed in clinical trials
(frequency ≥5% of subjects) were pyrexia, headache, cough,
nasopharyngitis, arthralgia, vomiting, upper respiratory tract
infection, limb injury, nasal congestion, and diarrhea.
Please see full prescribing information for ADVATE at:
www.advate.com /assets/pdf/advate_iri_pi.pdf.
About Baxalta
Baxalta Incorporated (NYSE: BXLT) is a $6
billion global biopharmaceutical leader developing,
manufacturing and commercializing therapies for orphan diseases and
underserved conditions in hematology, oncology and immunology.
Driven by passion to make a meaningful impact on patients' lives,
Baxalta's broad and diverse pipeline includes biologics with novel
mechanisms and advanced technology platforms such as gene therapy.
The Baxalta Global Innovation and R&D Center is located in
Cambridge, Massachusetts. Launched
in 2015 following separation from Baxter International Inc,
Baxalta's heritage in biopharmaceuticals spans decades. Baxalta's
therapies are available in more than 100 countries and it has
advanced biological manufacturing operations across 12 facilities,
including state-of-the-art recombinant production and plasma
fractionation. Headquartered in Northern Illinois, Baxalta employs 16,000
employees worldwide.
Forward-Looking Statements
This release includes forward-looking statements concerning
ADYNOVATE, including expectations with regard to clinical trials,
future regulatory actions, expected launch plans and potential
impact on patients. Such statements are made of the date that they
were first issued and are based on current expectations, beliefs
and assumptions of management. Forward-looking statements are
subject to a number of risks and uncertainties, many of which
involve factors or circumstances that are beyond Baxalta's control
and which could cause actual results to differ materially from
those in the forward-looking statements, including the following:
clinical trial results; satisfaction of regulatory and other
requirements; actions of regulatory bodies and other governmental
authorities; changes in laws and regulations; product quality,
manufacturing or supply issues; patient safety issues; and other
risks identified in Baxalta's Registration Statement on Form 10 and
other Securities and Exchange Commission filings, all of which are
available on Baxalta's website. Baxalta expressly disclaims
any intent or obligation to update these forward-looking statements
except as required by law.
Baxalta, Advate and Adynovate are trademarks of Baxalta
Incorporated.
All other product brands or trademarks
appearing herein are the property of their respective
owners.
References:
1. Proprietary pegylation technology
exclusively licensed from Nektar Therapeutics.
2. Konkle, Barbara, et al. Pegylated, full-length, recombinant
factor VIII for prophylactic and on- demand treatment of severe
hemophilia A. Blood. July 2015.
3. National Hemophilia Foundation. Fast Facts. Accessed
August 16, 2015.
https://www.hemophilia.org/About-Us/Fast-Facts.
Baxalta Media Relations
Kellie
Hotz, +1-224-940-2202, media@baxalta.com
Baxalta Investor Relations
Mary Kay Ladone, +1-224-948-3371,
mary.kay.ladone@baxalta.com
Lorna Williams, +1-224-948-3511,
lorna.williams@baxalta.com
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SOURCE Nektar Therapeutics