Baxter International has filed a biologics licence application (BLA) to the US Food and Drug Administration (FDA) seeking the approval of OBI-1, a recombinant antihaemophilic porcine sequence factor VIII, for treatment of adult patients with acquired haemophilia A.
The filing is based on a global, prospective, multi-centre Phase II/III open label clinical trial, which evaluated the efficacy and safety of OBI-1 in the treatment of serious bleeds in adults with acquired haemophilia A, which is a rare, potentially life-threatening bleeding disorder.
Unlike congenital haemophilia, acquired haemophilia A typically affects older adults and occurs equally in both males and females.
In the trial, patients (N=18) 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.
Primary efficacy endpoint of the trial was defined by clinical assessment as effective or partially effective control of bleeding and FVIII activity levels at 24 hours after the start of OBI-1therapy.
The company said that all patients in the trial responded positively (14 effective / 4 partially effective) in the first 24 hours, based on clinical assessment and FVIII activity levels.
Results of the trial were presented at the American Society of Hematology ‘s (ASH) 55th Annual Meeting in New Orleans, Louisiana, US.
Tulane University associate professor of medicine and pediatrics Rebecca Kruse-Jarres said the results showed that all patients in the trial experienced a positive response to treatment with OBI-1 within 24 hours of initiation of care.
"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," Kruse-Jarres added.
In the trial, no treatment-related serious adverse events were reported, but non-serious mild adverse events related to treatment were observed in two of 18 patients.
The company said that the two patients developed anti-porcine inhibitors to OBI-1, which has been granted orphan-drug designation for acquired hemophilia A by the FDA.