Baxter International Inc. (NYSE:BAX) today announced that the company
has submitted a biologics license application (BLA) to the United States
(U.S.) Food and Drug Administration (FDA) for the approval of BAX111,
the first highly-purified recombinant von Willebrand Factor (rVWF) in
clinical development as a treatment for patients with von Willebrand
disease, the most common type of inherited bleeding disorder.1
''If approved, BAX111 will be the first recombinant replacement
treatment for von Willebrand disease, offering an important new option
that may provide greater flexibility in treating patients with this
challenging disease,'' said John Orloff, vice president and global head
of research and development at Baxter BioScience. ''Filing for U.S.
approval for this treatment helps us further advance our pursuit of new
treatment options and improved quality of care for people with a range
of bleeding disorders around the world.''
The filing was based on the completion of a Phase III, multi-center,
open-label clinical trial assessing the safety, efficacy and
pharmacokinetics of BAX111. The study met its primary efficacy endpoint
defined by the number of patients who achieved treatment success for
control of bleeding episodes. All patients treated in the full analysis
set (n=22) experienced a 100% treatment success rating based on a
4-point efficacy rating scale, comparing prospectively estimated number
of infusions needed to treat the bleeding episodes to the actual number
of infusions administered. The median number of infusions required to
treat bleeding events in the trial was 1.0 and the majority of events
(81.1%) were resolved with a single infusion.
A total of 125 adverse events (AE’s) following 318 infusions occurred in
25/37 subjects. Eight AEs were considered causally related to BAX111:
six non-serious related AEs (tachycardia, infusion site paresthesia,
electrocardiography (ECG) T-wave inversion, dysgeusia, generalized
pruritis and hot flush) occurred in four patients, and two related SAEs
(chest discomfort and increased heart rate) occurred in one patient.
Baxter expects to publish additional data from the trial in the coming
months. Both the European Commission and the U.S. Food and Drug
Administration have granted orphan-drug designation for BAX111.
About von Willebrand Disease2,3
Von Willebrand Disease (VWD) is an autosomal genetic disorder related to
quantitative deficits and/or qualitative defects of VWF, the result of
which is impaired hemostasis. It is the most common hereditary
coagulation disorder, occurring in approximately one to two percent of
the general population. Many people who have VWD may experience mild
symptoms, but some patients can experience severe bleeding events
similar to bleeding experienced by patients with hemophilia.
About Baxter BioScience
Baxter BioScience is a leading provider of therapeutic treatments that
save, sustain and improve the lives of people with rare conditions,
chronic diseases or limited treatment options. Supported by advanced
technical and manufacturing expertise, Baxter BioScience has a broad
pipeline built on a legacy of innovation in bleeding disorders and
immunology and is expanding to address emerging opportunities in niche
areas of oncology as well as technology platforms such as biosimilars.
About Baxter International Inc.
Baxter International Inc., through its subsidiaries, develops,
manufactures and markets products that save and sustain the lives of
people with hemophilia, immune disorders, cancer, infectious diseases,
kidney disease, trauma and other chronic and acute medical conditions.
As a global, diversified healthcare company, Baxter applies a unique
combination of expertise in medical devices, pharmaceuticals and
biotechnology to create products that advance patient care worldwide.
This release includes forward-looking statements concerning BAX111,
including expectations with regard to the potential impact of BAX111 to
patients. The statements are based on assumptions about many important
factors, including the following, which could cause actual results to
differ materially from those in the forward-looking statements:
satisfaction of regulatory and other requirements; actions of regulatory
bodies and other governmental authorities; additional clinical results;
changes in laws and regulations; product quality, manufacturing or
supply, or patient safety issues; and other risks identified in Baxter's
most recent filing on Form 10-K and other SEC filings, all of which are
available on Baxter's website. Baxter does not undertake to update its
forward-looking statements.
1. Laffan MA, Lester W, O'Donnell JS, et al. The diagnosis and
management of von Willebrand disease: a United Kingdom Haemophilia
Centre Doctors Organization guideline approved by the British Committee
for Standards in Haematology. Br J Haematol. 2014; 167: 453–465.
2. Nichols WL, Hultin MB, James AH, et al. Guidelines. von Willebrand
disease (VWD): evidence-based diagnosis and management guidelines, the
National Heart, Lung, and Blood Institute (NHLBI) Expert Panel report
(USA). Haemophilia. 2008; 14: 171–232.
3. Mannucci PM. Treatment of von Willebrand's disease. N Eng J Med.
2004; 351: 683–694.
Copyright Business Wire 2014