Baxter International has submitted a biologics license application (BLA) to the US Food and Drug Administration (FDA) for the approval of a recombinant antihemophilic porcine sequence factor VIII called 'OBI-1' for the treatment of patients with acquired hemophilia A.
The submission is based on a global, prospective, multi-center Phase II/III open label clinical trial, which assessed the efficacy and safety of OBI-1 in the treatment of serious bleeds in adults with acquired hemophilia A.
During the trial, patients (N=18) with a serious bleed were administered 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 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.
According to the company, 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.
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 said.
The company said that no treatment-related serious adverse events were reported in the trial, but non-serious mild adverse events related to treatment were observed in two of 18 patients, who developed anti-porcine inhibitors to OBI-1.
