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Twice-Weekly Factor VIII Treatment Prevents Bleeding in Hemophilia Patients
Positive data from a phase II/III pivotal trial and a phase I study of BAX 855 (Baxalta Incorporated) have been published in Blood, the journal of the American Society of Hematology. BAX 855 is an investigational, extended half-life recombinant factor VIII (rFVIII) treatment for hemophilia A based on Advate (antihemophilic factor [recombinant], Baxter), a leading treatment for hemophilia A with more than 11 years of real-world patient experience.
The publication provides an overview of the clinical trial results for BAX 855, which will be marketed in the U.S. under the brand name Adynovate (antihemophilic factor [recombinant], pegylated) upon approval. The positive study results supported Baxalta’s submission of BAX 855 to the FDA in December 2014.
The prospective, global, open-label, two-arm, phase II/III study evaluated BAX 855 in 137 previously treated patients aged 12 to 65 years with hemophilia A. The patients were assigned to receive either twice weekly prophylaxis (40 to 50 IU/kg; n=120) or on-demand treatment (10 to 60 IU/kg; n = 17). BAX 855 met the study’s primary endpoint for the prevention of bleeding episodes. Patients in the twice-weekly prophylaxis arm of the trial experienced a 95% reduction in the median annualized bleed rate compared with those in the on-demand arm (1.9 vs. 41.5, respectively). BAX 855 was also effective in treating all bleeding episodes, 95.9% of which were controlled with one or two infusions at a median dose of 29.0 IU/kg per infusion.
Treatment with BAX 855 was rated excellent or good for nearly all bleeding episodes (96.1%). In the prophylactic group (n = 101), 39.6% of compliant patients experienced no bleeds. The study also showed that BAX 855 pharmacokinetics offered a 1.4- to 1.5-fold mean extended half-life compared with Advate, with a median infusion interval of 3.6 days. No patients developed inhibitors to BAX 855, and no treatment-related serious adverse events, including hypersensitivity reactions, were reported. Adverse events included headache, diarrhea, nausea, and flushing.
Globally, an estimated 70% of people with hemophilia remain undiagnosed and untreated or undertreated.
Source: Baxalta; July 16, 2015.