DEERFIELD (dpa-AFX) - Baxter International Inc. (BAX) announced that the United States Food and Drug Administration or FDA has approved Obizur [Antihemophilic Factor (Recombinant), Porcine Sequence] for the treatment of bleeding episodes in adults with acquired hemophilia A or AHA, a very rare and potentially life-threatening acute bleeding disorder.
The company stated that Obizur will be commercially available in the United States in the coming months and is currently under regulatory review in Europe and Canada.
Obizur is the first recombinant porcine FVIII treatment approved for AHA that allows physicians to manage the treatment's efficacy and safety by measuring factor VIII activity levels in addition to clinical assessments. Obizur replaces the inhibited human factor VIII with a recombinant porcine sequence factor VIII based on the rationale that it is less susceptible to inactivation by circulating human factor VIII antibodies.
The approval is based on a global, prospective, controlled, multi-center Phase 2/3 open-label clinical trial that examined the efficacy of Obizur in the treatment of serious bleeding episodes in adults with AHA (29 patients evaluated for safety, 28 evaluated for efficacy). All patients treated with Obizur (28/28) showed a positive response, meaning an effective or partially effective response with bleeding stopped or reduced and clinical improvement, at 24 hours after the initial infusion.
A total of 86 percent (24/28) had successful treatment of the initial bleeding episode. Common adverse reactions observed in greater than five percent of 29 patients in the clinical trial were development of inhibitors to porcine FVIII.
Obizur was granted orphan-drug status by the FDA and its review was prioritized based on AHA's classification as a rare disease and the potential for the treatment to address an important unmet medical need.
Acquired hemophilia A is a rare, potentially life-threatening bleeding disorder, which, unlike congenital hemophilia, typically affects older adults and occurs in both males and females. In acquired hemophilia A, individuals typically experience subcutaneous, soft tissue, and post-surgical bleeding. The comorbidities in this typically elderly population also pose a particular challenge to treat serious bleeding episodes.
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