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FDA approves Obizur for acquired hemophilia A
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The FDA approved Obizur for the treatment of bleeding disorders in adults with acquired hemophilia A.
The development of acquired hemophilia A — a non-genetic Factor VIII deficiency — has been associated with pregnancy, cancer and the use of certain medications, but about half of cases originate from unknown causes.
Obizur (Antihemophilic Factor [Recombinant], Porcine Sequence; Baxter Healthcare] treats bleeding episodes in these patients by replacing the inhibited human Factor VIII with a recombinant porcine sequence Factor VIII.
Obizur is the first treatment of its kind and enables researchers to measure Factor VIII activity levels in patients with the disease. The FDA previously granted orphan drug designation to the agent due to its ability to treat a rare disease.
Karen Midthun
“The approval of this product provides an important therapeutic option for use in the care of patients with this rare disease,” Karen Midthun, MD, director of FDA’s Center for Biologics Evaluation and Research, said in a press release issued by the FDA.
The FDA based its approval of Obizur in part on the results of an open-label, phase 2/phase 3 trial that included 29 patients.
Of the 28 patients evaluable for efficacy, 100% demonstrated an effective or partially effective response with stopped or reduced bleeding within 24 hours after the initial infusion. Researchers also reported 86% of patients had successful treatment — defined as the ability to discontinue treatment or reduce the dose or dosing frequency — of the initial bleeding episode.
A common adverse event that occurred in more than 5% of all patients on the trial was the development of inhibitors to porcine Factor VIII.
“The approval of Obizur is welcome news for the hemophilia community based on the data from the first clinical trial designed specifically for acquired hemophilia A, which found that all patients responded to treatment within 24 hours,” researcher Rebecca Kruse-Jarres, MD, director of the Hemophilia Care Program at Puget Sound Blood Center in Seattle, said in a press release issued by Baxter. “Importantly, this new option to treat bleeding episodes will enable us to measure Factor VIII levels, thus giving us an objective marker of hemostasis that can guide dosing and prevent overdosing.”