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Hemophilia Drug BAX 855 Submitted for FDA Review
A biologics license application (BLA) has been submitted to the FDA for BAX 855 (Baxter International, Inc.), an investigational, extended-half-life recombinant factor VIII treatment for hemophilia A based on Advate (antihemophilic factor [recombinant], Baxter).
The submission was based on positive results from a prospective, global, open-label, two-arm, phase III study in 137 previously treated patients with hemophilia A. BAX 855 met the trial’s primary endpoint in the control and prevention of bleeding episodes and in routine prophylaxis for patients aged 12 years or older.
Patients in the twice-weekly prophylaxis arm experienced a 95% reduction in the median annual bleed rate (ABR) compared with those in the on-demand arm (1.9 vs. 41.5, respectively). BAX 855 was also effective in treating bleeding episodes, 96% of which were controlled with one or two infusions. No patients developed inhibitors to BAX 855, and no treatment-related serious adverse events, including hypersensitivity, were reported. The most common product-related adverse reaction was headache, which occurred in three patients.
BAX 855 is based on Advate (Baxter), a full-length FVIII molecule with more than 10 years of real-world experience. Advate is currently approved in 64 countries worldwide, including the U.S. and Canada, for use in children and adults with hemophilia A (congenital factor VIII deficiency or classic hemophilia). It is not indicated for the treatment of von Willebrand disease.
Source: Baxter International, Inc.; December 1, 2014.