Bayer Healthcare announced that it has submitted a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for BAY 81-8973, a recombinant Factor VIII (rFVIII) compound, for the treatment of hemophilia A in children and adults.

The BLA submission was based on results from the LEOPOLD (Long-Term Efficacy Open-Label Program in Severe Hemophilia A Disease) clinical trials, which evaluated BAY 81-8973 in adults and children using two- and three-times per week prophylaxis dosing regimens.

LEOPOLD I is an open-label, randomized study. Part A studied the pharmacokinetics of BAY 81-8973. Part B evaluated the safety, tolerability and efficacy of prophylaxis with BAY 81-8973. Part C evaluated hemostatic outcome during major surgery. Participants ages 12–65 years with severe hemophilia A (<1% FVIII:C) were included. The primary endpoint of Part B was annualized number of all bleeds over 12 months.

RELATED: Investigational Hemophilia A Therapy BLA Submitted by Baxter

LEOPOLD II is a Phase 2/3 randomized, cross-over, open-label trial also in males aged 12–65 years. Participants were randomized to receive BAY 81-8973 either as a low-dose prophylaxis regimen (20–30 IU/kg; n=28) twice-per-week, high-dose prophylaxis (30–40 IU/kg; n=31) three-times-per-week, or on-demand (n=21). The primary objective was to demonstrate the superiority of prophylaxis vs. on-demand therapy, with the primary endpoint being bleeding frequency at 12 months.

LEOPOLD Kids is a two-part, open-label, non-randomized Phase 3 study. Part A is designed to evaluate the efficacy and safety of BAY 81-8973 for prophylaxis, treatment of bleeds, and surgical management in previously treated children ≤12 years of age. Part B of the study is ongoing.

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