Introduction:
BAY 81-8973 is Bayer’s new full-length recombinant factor VIII (FVIII) product in development for the treatment of hemophilia A. BAY 81-8973 has no human- or animal- derived raw materials added to the cell culture, purification, or formulation processes.
Objective:
To evaluate the safety profile of BAY 81-8973 as documented in clinical trials with BAY 81-8973.
Methods:
The safety of BAY 81-8973 for prevention and treatment of bleeds in patients with severe hemophilia A (<1% FVIII) was evaluated in 3 clinical studies: 2 in adolescent and adult previously treated patients (PTPs; aged 12–65 years with ≥150 previous exposure days [EDs]) and 1 in pediatric PTPs (aged ≤12 years with ≥50 previous EDs). A total of 193 PTPs (including 51 pediatric patients) were included to assess the frequency of adverse reactions in the 3 phase 3 studies. The immunogenicity of BAY 81-8973 was also evaluated in PTPs. Adverse reactions were collected and analyzed throughout the studies.
Results:
The frequency, type, and severity of adverse reactions in children were similar to those in adults. Adverse reactions in PTPs are listed in Table 1. In PTPs evaluated to date for immunogenicity, no inhibitors were detected.
Table 1. Adverse Reactions in Previously Treated Patients (N=193)
Conclusions:
The incidence of treatment-related adverse and serious adverse reactions in the BAY 81-8973 trials was low (<10%) and similar to those reported in the literature for other full-length FVIII products. No PTP developed a FVIII inhibitor.