BANNOCKBURN, Ill., Dec. 21, 2015 /PRNewswire/ -- Baxalta
Incorporated (NYSE: BXLT), a global biopharmaceutical leader
dedicated to delivering transformative therapies to patients with
orphan diseases and underserved conditions, today announced initial
results from a Phase 3 clinical trial of
ADYNOVATE [Antihemophilic Factor
(Recombinant), PEGylated], an
extended circulating half-life recombinant Factor VIII
(rFVIII) treatment for hemophilia A based on ADVATE [Antihemophilic
Factor (Recombinant)]. ADYNOVATE was approved by the U.S. Food and
Drug Administration (FDA) in November
2015 for use in adolescent and adult hemophilia patients (12
years and older) for on-demand treatment and control of bleeding,
and prophylaxis to reduce the frequency of bleeding
episodes.
The prospective, uncontrolled, open-label, multi-center Phase 3
study was designed to assess the safety and immunogenicity of
ADYNOVATE. The study enrolled 73 previously-treated patients (PTPs)
with severe hemophilia A younger than 12 years of age and assessed
the treatment's hemostatic efficacy in prophylaxis and treatment of
bleeding episodes. All participants received prophylactic ADYNOVATE
treatment (median 1.9 infusions per week) and were followed for six
months.
ADYNOVATE met its primary endpoint in the study, as 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 and nearly 40 percent (37.9 percent)
experienced zero bleeds. The median 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.
"These initial efficacy and safety findings indicate a
potentially valuable role for ADYNOVATE to treat pediatric patients
with hemophilia A, with data consistent with what was reported in
clinical studies among adult patients," said John Orloff, M.D., head of Research &
Development and chief scientific officer, Baxalta. "We will
continue to build evidence on the value of ADYNOVATE through our
robust clinical development program, which will support additional
global registrations in the coming years."
With the study results, the company plans to file for marketing
authorization in Europe and aims
to file for a pediatric indication in the U.S. in early 2016.
ADYNOVATE is currently under regulatory review in Japan,
Canada and Switzerland. Baxalta plans to present the
complete data from this study at a congress in 2016.
In addition to an ongoing study in the surgical setting,
Baxalta's continuation study remains ongoing to assess long-term
safety and efficacy in PTPs with severe hemophilia A. The company
has also recently announced the initiation of a study of
previously-untreated patients (PUPs) with severe hemophilia A, as
well as a study to evaluate pharmacokinetic (PK)-guided prophylaxis
dosing with ADYNOVATE (the PROPEL study).
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
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.
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.
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.
Please see full prescribing information for ADVATE
at: http://www.baxalta.com/assets/documents/ADVATE_PI.pdf.
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.
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 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 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, Advate and Adynovate are trademarks of Baxalta
Incorporated.
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