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. 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.
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.''
Baxter International Inc.