Baxalta’s ADYNOVATE Meets Endpoints in Phase 3 Study of Hemophilia A Patients Under 12 Years of Age

  • Topline results find no inhibitors to ADYNOVATE [Antihemophilic
    Factor (Recombinant), PEGylated], treatment were reported and nearly
    73 percent of treated patients experienced zero joint bleeds
  • ADYNOVATE approved for adults in November 2015, offers proven bleed
    prevention with a simple, twice-weekly dosing schedule
  • These data will form the basis of a U.S. filing for a pediatric
    indication in early 2016

BANNOCKBURN, Ill.–(BUSINESS WIRE)–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.


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)

  • 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


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).


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


Common adverse reactions (≥1% of subjects) reported in the clinical
studies were headache and nausea.

For Full Prescribing Information, visit


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

ADVATE is not indicated for the treatment of von Willebrand disease.

Detailed Important Risk Information


ADVATE is contraindicated in patients who have life-threatening
hypersensitivity reactions, including anaphylaxis, to mouse or hamster
protein or other constituents of the product.


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

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.


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:

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
Investor Relations

Mary Kay Ladone, +1-224-948-3371
Williams, +1-224-948-3511