On October 24, 2014, the US Food and Drug Administration (FDA) approved Obizur [Antihemophilic Factor (Recombinant) Porcine Sequence] to treat bleeding episodes in adults with acquired hemophilia A. Porcine FVIII is comparable enough to human FVIII to effectively stem bleeding, while less likely to trigger an unwanted immune response. The product is manufactured by Baxter Healthcare.
A rare though potentially life-threatening disorder, the onset of acquired hemophilia A is often associated with other medical conditions or changes in health status that elicit the production of antibodies by the immune system. Conditions most often linked to acquired hemophilia A include cancers, pregnancy and the use of particular medications. In approximately half of reported cases, the cause is unknown.
Bleeds in these patients typically occur in the skin, muscles and soft tissue, rather than the joints.
A clinical trial including 29 adults with acquired hemophilia A was the basis for demonstrating the safety and efficacy of Obizur. Of the test group, 86% had successful treatment after initial infusion to treat a bleeding episode. However, more than 5% of subjects developed an inhibitor to the product. Obizur cannot be used in patients with a known hypersensitivity reaction to hamster proteins.
“The approval of this product provides an important therapeutic option for use in the care of patients with this rare disease,” said Karen Midthun, MD, director of FDA’s Center for Biologics Evaluation and Research.
Obizur is under regulatory review in Canada and Europe.
Source: FDA news release dated October 24, 2014.
