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FDA
APPROVES BAXTER'S ADVATE FOR THE TREATMENT OF HEMOPHILIA A
Deerfield, Ill., July
25, 2003 -- Baxter Healthcare Corporation announced today that the U.S.
Food and Drug Administration (FDA) has approved ADVATE (Antihemophilic
Factor (Recombinant), Plasma/Albumin-Free Method) rAHF-PFM for the prevention
and control of bleeding episodes in people with hemophilia A. ADVATE is
the first and only factor VIII made without any added human or animal
plasma proteins and albumin in the cell culture process, purification
and final formulation, thereby eliminating the risk of infections caused
by viruses that may be carried in these proteins.
Developed by Baxter through its innovative technology and many years in
hemophilia research, ADVATE is a genetically engineered, or recombinant,
full-length clotting factor VIII that is similar to the natural clotting
factor found in a healthy body. The new technology used to prepare ADVATE
- which is the most advanced factor VIII treatment for hemophilia to date
- eliminates the addition of human and animal plasma proteins and albumin
to provide unsurpassed pathogen safety, while maintaining the proven efficacy
of the full-length RECOMBINATE rAHF molecule, the current leader for hemophilia
A therapy.
"It's widely
recognized by the hemophilia community that there is a need to provide
patients with treatments that eliminate the risk for infection. While
treatment options to date have provided effective disease management,
there has been an acknowledged, lingering concern about the potential
for viral transmission with these therapies," said Michael Tarantino,
M.D., associate professor of Pediatrics at the University of Illinois
and medical director of the Comprehensive Bleeding Disorder Center in
Peoria, Ill. "ADVATE is the first therapy to meet this need for safety,
virtually eliminating the risk of transmission of human pathogens."
Other genetically
engineered factor VIII therapies use human plasma protein solutions and/or
other human- or animal-derived materials during processing, or add human
albumin for stabilization. These components have the potential to carry
disease-causing viruses or pathogens, such as HIV, West Nile Virus or
variant Creutzfeldt-Jakob Disease (vCJD), the human form of mad cow disease.
The Medical and Scientific Advisory Council (MASAC) of the National Hemophilia
Foundation has issued formal recommendations (#141) advising that "all
efforts should be made to remove human albumin from recombinant factor
VIII products" and " increased efforts should be made to eliminate
human and bovine proteins from the manufacturing process of recombinant
products." ADVATE is the first and only FDA-approved factor VIII
therapy to meet these guidelines.
"During the last
40 years, Baxter has strived continually to provide the hemophilia community
with enhanced, safer factor VIII therapeutics. The FDA approval of ADVATE
marks another important milestone for Baxter and the hemophilia community,"
said Thomas Glanzmann, president, Baxter BioScience. "Baxter is pleased
to have consistently met the needs of the hemophilia community by introducing
innovative technology to elevate the standard of care."
ADVATE CLINICAL
DATA
Phase II-III clinical
trials evaluated the safety, immunogenicity and efficacy of ADVATE in
the treatment and prevention of bleeding in previously treated hemophilia
patients in a variety of clinical settings. Data demonstrate that ADVATE
has equivalent pharmacokinetic properties to RECOMBINATE, and is effective
in controlling bleeding episodes, which can occur as often as three times
per month in patients with severe hemophilia A.
"The development
of ADVATE is based on our scientific expertise and application of innovative
technologies to advance the treatment of hemophilia," said Norbert
Riedel, Ph.D., chief scientific officer, Baxter. "ADVATE is the first
and only factor VIII therapy that combines the unsurpassed pathogen safety
of a plasma/albumin-free method with the proven efficacy of the RECOMBINATE
molecule."
Side effects with
the use of ADVATE are uncommon, with no serious adverse events related
to treatment with ADVATE reported to date. Non-serious, moderate or mild
adverse events related to ADVATE include: a strange taste, itching at
the infusion site, dizziness, headaches, catheter-related infections,
chills, hot flashes, diarrhea, lower limb edema, sweating, nausea, pain
in the upper abdomen or lower chest, prolonged bleeding after post-operative
drain removal, decreased red blood cell count, joint swelling and shortness
of breath. Non-serious, but severe related adverse events include: fever,
headache and decreased coagulation factor VIII levels.
ABOUT ADVATE
ADVATE is a blood-clotting
therapy that helps people with hemophilia A prevent and control bleeding
episodes. Infused directly into the bloodstream, ADVATE works by temporarily
raising the level of factor VIII in the blood, thus allowing the body's
blood clotting process to function properly. Baxter expects to begin shipping
ADVATE to distributors in 3-6 weeks.
ADVATE is also convenient
to use because it is available in super-high potency (1500 IU/vial), with
a smaller infusion volume (5mL diluent), which means shorter infusion
times for patients. ADVATE comes packaged with a 10mL syringe, butterfly
bandage, alcohol swab and a patient-friendly package insert.
ADVATE is processed
by Baxter in its state-of-the art, multi-purpose, biotechnology facility
in Neuchâtel, Switzerland. This facility has the capacity to help
ensure sufficient supplies of this therapy based on current and anticipated
patient needs.
ADVATE is under regulatory
review in Europe and Canada following Baxter's submission of a Marketing
Authorization Application to the European Medicines Evaluation Agency
through the European Union Centralized Procedure, and a New Drug Submission
with Health Canada's Drug's Directorate in September 2002.
ABOUT HEMOPHILIA
People with hemophilia
A do not produce adequate amounts of factor VIII, which is necessary to
effectively clot blood. Without enough factor VIII, patients can experience
spontaneous, uncontrolled internal bleeding that is painful, debilitating
and damaging to joints. If untreated, patients with severe hemophilia
A have a greatly reduced life expectancy.
According to the World
Health Organization, more than 400,000 people in the world may have hemophilia
A and it affects 15-20 of every 100,000 males born worldwide. People with
hemophilia A infuse themselves with clotting factors three times a month,
on average, unless they are on a preventive regimen, in which case their
physician may prescribe infusing a therapy as often as three or four times
per week.
ABOUT BAXTER
Baxter Healthcare
Corporation is the principal domestic operating subsidiary of Baxter International
Inc. (NYSE: BAX). Baxter International Inc., through its subsidiaries,
assists health-care professionals and their patients with treatment of
complex medical conditions, including cancer, hemophilia, immune disorders,
kidney disease and trauma. The company applies its expertise in medical
devices, pharmaceuticals and biotechnology to make a meaningful difference
in patients' lives.
Questions and Answers
What are the labeling
differences between RECOMBINATE and ADVATE?
ADVATE is derived from the same full-length molecule as RECOMBINATE and,
therefore, has a similar label and indication. However, ADVATE offers
greater convenience based on its higher potency and lower infusion volume,
as well as the safety of its innovative plasma/albumin-free process.
Will ADVATE be
priced higher than RECOMBINATE?
ADVATE is priced slightly higher than RECOMBINATE, but within the range
of existing recombinant factor VIII therapies. This is based on the fact
that ADVATE is the most advanced hemophilia therapy available, combining
unsurpassed pathogen safety with the proven efficacy of the RECOMBINATE
molecule. ADVATE was also developed using complex, innovative technology.
Are you expecting
any large initial stocking orders?
We are in discussions with our customers and home care companies to ensure
we provide them with the product necessary to meet the needs of their
patients.
What do you anticipate
will drive ADVATE sales in the U.S.?
Based on its many advantages, we believe ADVATE will appeal to a broad
spectrum of customers. We anticipate the key drivers for ADVATE use to
include patients switching from RECOMBINATE, patients switching from plasma-based
products and patients switching from competitive products.
Do you anticipate
patients will switch to ADVATE from RECOMBINATE or from competitor's products?
We anticipate many patients will switch to ADVATE regardless of what their
current therapy may be. The hemophilia community has indicated there will
be a high demand for this innovative therapy because it removes concerns
about unknown viruses and infectious proteins carried in protein additives.
What are your expected
recombinant sales (ADVATE and RECOMBINATE combined) in 2003?
We expect that Baxter's global recombinant sales (from both ADVATE and
RECOMBINATE) will grow more than 10 percent in 2003.
What is the size
of the global recombinant marketplace?
The current recombinant marketplace is approximately 1.9 billion activity
units. We expect this market to grow to 2.5-2.9 billion units by 2005
based on continued increases in availability and promotion of recombinant
products. The three key drivers of the recombinant market are conversion
from plasma-derived therapies, adequate dosing and compliance, and increasing
prophylactic use of these therapies.
When do you expect
European approval?
We continue to anticipate European approval in the second half of this
year.
What clinical studies
are included in the development program for ADVATE?
There are seven clinical studies in the development program for ADVATE:
- Pivotal
Study in Previously Treated Patients - data presented at the
American Society of Hemotology Annual Meeting, December, 2002
- Continuation
Study of Previously Treated Patients - (ongoing) preliminary
data presented at the International Society for Thrombosis and Haemostasis
(ISTH) meeting, July 2003
- Surgery
Study - (ongoing) preliminary data presented at the ISTH meeting,
July 2003
- Pediatric
Study - (ongoing) preliminary data presented at the ISTH meeting,
July 2003
- Study in
Japanese Patients - required for regulatory submission in Japan
Baxter also plans
to initiate a study in previously untreated patients and also has plans
for additional Phase IV studies.
Baxter, ADVATE,
and RECOMBINATE are trademarks of Baxter International Inc. and its affiliates.
Please see full prescribing
information or visit www.advate.com
for further information.
This news release
contains forward-looking statements that involve risks and uncertainties,
including technological advances in the medical field, the effect of economic
conditions, actions by regulatory authorities at any stage of the development
and commercialization process, product demand and market acceptance, the
impact of competitive products and pricing, and other risks detailed in
the company's filings with the Securities and Exchange Commission. These
forward-looking statements are based on estimates and assumptions made
by management of the company and are believed to be reasonable, though
are inherently uncertain and difficult to predict. Actual results or experience
could differ materially from the forward-looking statements.
FOR ADDITIONAL
INFORMATION:
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For Baxter
- Media Contacts:
- Cindy Resman,
847-948-2815
Deborah Spak, 847-948-2349
- Investor Contacts:
- Neville Jeharajah,
847-948-2875
Mary Kay Ladone, 847-948-3371
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