FDA approves Obizur for treatment of Haemophilia A patients- Baxter

Baxter International Inc.announced that the FDA has approved Obizur [Antihemophilic Factor (Recombinant), Porcine Sequence] for the treatment of bleeding episodes in adults with acquired Hemophilia A (AHA), a very rare and potentially life-threatening acute bleeding disorder. 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. 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 II/III 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. The overall treatment success was determined by the investigator based on the ability to discontinue or reduce the dose and/or dosing frequency of Obizur. Common adverse reactions observed in greater than five percent of 29 patients in the clinical trial were development of inhibitors to porcine FVIII.