DEERFIELD, Ill., December 10, 2013 - Baxter International Inc. (NYSE:BAX) today announced that the company has submitted a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) for the approval of OBI-1, a recombinant antihemophilic porcine sequence factor VIII, in patients with acquired hemophilia A. Phase 2/3 data supporting the submission were presented at the American Society of Hematology's (ASH) 55th Annual Meeting in New Orleans, LA.
"The data we presented today at ASH showed that all patients in the trial experienced a positive response to treatment with OBI-1 within 24 hours of initiation of care," said Rebecca Kruse-Jarres, M.D., MPH, Associate Professor of Medicine and Pediatrics, Tulane University and past National Hemophilia Foundation-Baxter Fellow. "These are promising results for a patient population that would benefit from a treatment option that provides temporary FVIII replacement and measurement of FVIII levels."
The global, prospective, multi-center phase 2/3 open label clinical trial examined the efficacy and safety of OBI-1 in the treatment of serious bleeds in adults with acquired hemophilia A. Patients (N=18) who presented with a serious bleed were treated with an initial dose of OBI-1 (200 units per kilogram), followed by additional doses based on their personal profiles, including clinical evaluations and target factor VIII activity levels. The primary efficacy endpoint of the study was defined by clinical assessment as effective or partially effective control of bleeding and FVIII activity levels at 24 hours after initiation of OBI-1therapy. All patients in the study responded positively (14 effective / 4 partially effective) in the first 24 hours, based on clinical assessment and FVIII activity levels.
No treatment-related serious adverse events were reported in the study. Non-serious mild adverse events related to treatment were reported in two of 18 patients (11.1%). These two patients developed anti-porcine inhibitors to
OBI-1 has been granted orphan-drug designation for acquired hemophilia A by the FDA. Orphan-drug designation is given to drugs and biologics intended to treat rare diseases, typically those that impact less than 200,000 Americans annually. The application has also been granted a fast-track designation, which is intended to expedite the review process of drug candidates with the potential to fulfill an unmet medical need. Baxter acquired OBI-1 in March from Inspiration BioPharmaceuticals, Inc. and Ipsen Pharma S.A.S.
"Completing the BLA submission for OBI-1 in nine months following Baxter's acquisition of the molecule reflects our continuing commitment to advancing the care of hemophilia patients and extending our expertise and leadership in this category," said Ludwig Hantson, Ph.D., president of Baxter's BioScience organization. "This program has the potential to bring a new treatment option to the acquired hemophilia patient population, which has limited treatment options available today."
About Acquired Hemophilia A
Acquired hemophilia A is a rare, potentially life-threatening bleeding disorder, which, unlike congenital hemophilia, typically affects older adults and occurs equally in both males and females. In acquired hemophilia A, individuals typically experience subcutaneous, deep tissue, and post-surgical bleeding, in contrast to bleeding into joints, which is more typical in congenital hemophilia. Also, the comorbidities in this population provide a particular challenge in the treatment of serious bleeding episodes.
About Baxter in Hemophilia
Baxter has more than 60 years experience in hemophilia and has introduced a number of therapeutic firsts for hemophilia patients. Baxter has the broadest portfolio of hemophilia treatments in the industry and is able to meet individual therapy choices, providing a range of options at each treatment stage. The company's work is focused on optimizing hemophilia care and improving the lives of people living with hemophilia A and B worldwide.
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 OBI-1, including expectations with regard to its potential impact on 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 approval and other requirements; actions of regulatory bodies and other governmental authorities; product quality or patient safety issues; changes in law and regulations; 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.