BANNOCKBURN, Ill., Feb. 25, 2016 /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 it
has submitted supplemental Biologics License Applications (sBLAs)
to the U.S. Food and Drug Administration (FDA) seeking
approval for the use of ADYNOVATE [Antihemophilic Factor
(Recombinant), PEGylated] to treat children under the age of
12 with hemophilia A and for use in surgical settings.
ADYNOVATE is the only extended circulating half-life
recombinant Factor VIII (rFVIII) treatment for hemophilia A based
on the demonstrated efficacy of ADVATE [Antihemophilic Factor
(Recombinant)]. ADYNOVATE was approved by the
FDA in November 2015 for use in adolescent and adult
hemophilia A patients (12 years and older) for prophylaxis to
reduce the frequency of bleeding episodes and on-demand treatment
and control of bleeding.
"We know there is a great need for innovative new treatments
among pediatric patients and during surgery and we look forward to
working with the FDA as they review ADYNOVATE for these
patients," said Brian Goff, executive vice president and
president, Hematology. "We are committed to continually advancing
direct factor replacement treatments and specifically to expanding
use of ADYNOVATE, providing as many patients as possible access to
its proven prophylaxis and simple, twice-weekly dosing
schedule."
The submission of ADYNOVATE to treat children under the age of
12 was based on results of a Phase 3 trial designed to assess the
efficacy and safety including immunogenicity of ADYNOVATE, which
was initially reported in December 2015. Results from the
study showed ADYNOVATE met its primary endpoint and no patients
developed inhibitory antibodies to ADYNOVATE. In addition, no
treatment-related serious adverse events were reported. More than
70 percent (72.7 percent) of patients had no joint bleeds while on
treatment with ADYNOVATE (n=66) and nearly 40 percent (37.9
percent) experienced zero bleeds. The median overall annualized
bleeding rate (ABR) among patient participants treated with
ADYNOVATE was 2.0 (range 0-49.8; mean ABR 3.0), which was
comparable to the rates seen in the adult study.
The filing was also supported by the positive results of a Phase
3 study evaluating the efficacy and safety of ADYNOVATE for the
perioperative control of hemostasis among 15 patients with severe
hemophilia A undergoing surgical procedures, which
was reported in December 2015. The study data demonstrated
that ADYNOVATE achieved hemostasis control in the perioperative
period (from start of the procedure until discharge or day 14) for
patients with severe hemophilia A.
Baxalta continues to invest in ADYNOVATE to expand the
product's value for more patients
worldwide. Baxalta plans to file for marketing
authorization in Europe in the first quarter of 2016 and
expects regulatory approval of the treatment in Japan in the
first half of the year. ADYNOVATE is also under regulatory review
in Canada and Switzerland.
ADYNOVATE is built on the full-length ADVATE molecule,
a leading treatment for hemophilia A that been used by patients
worldwide for more than 12 years. Through a collaboration
with Nektar Therapeutics (NASDAQ: NKTR), ADYNOVATE
leverages proprietary PEGylation technology designed to extend 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 time at which the body clears ADYNOVATE,
resulting in an extended 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.
About ADYNOVATE
Indications:
ADYNOVATE 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 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 67 countries worldwide, including
the United States, Canada, 28 countries in the European Union,
Algeria, Argentina, Australia, Brazil, Brunei, Chile, China,
Colombia, Ecuador, Hong
Kong, Iceland, India, Iraq,
Israel, Japan, Kuwait, Macau, Malaysia, Mexico, Morocco, New
Zealand, Norway,
Panama, Puerto Rico, Qatar, Russia, Saudi
Arabia, Serbia, Singapore,
South Korea, Suriname,
Switzerland,Taiwan, Tunisia,
Turkey, Ukraine, Uruguay, and Venezuela.
Indications:
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.
Detailed Important Risk Information
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.
For full prescribing information for
ADVATE, visit:
http://www.baxalta.com/assets/documents/ADVATE_PI.pdf.
About Baxalta
Baxalta Incorporated (NYSE: BXLT) is a global
biopharmaceutical leader developing, manufacturing and
commercializing therapies for orphan diseases and underserved
conditions in hematology, immunology and oncology. 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. Launched in
2015 following separation from Baxter International, 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, with its Global Innovation
Center in Cambridge,
Mass., Baxalta employs 17,000 employees worldwide.
Forward-Looking Statements
This release includes forward-looking statements concerning
ADYNOVATE, including expectations with regard to regulatory filings
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: 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 filings with
the Securities and Exchange Commission, 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 Media Relations
Kellie
Hotz, 1-224-940-2202
media@baxalta.com
Lauren Denz, 1-224-940-3259
lauren.denz@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