BAY 94-9027 is a B-domain-deleted prolonged-half-life recombinant factor VIII (FVIII) conjugates in a site-specific manner with polyethylene glycol.
Assess efficacy and safety of BAY 94-9027 for prophylaxis and treatment of bleeds in patients with severe hemophilia A PATIENTS/METHODS: In this multinational, phase 2/3, partially randomized, open-label trial, males aged 12-65 years with FVIII <1% and ≥150 exposure days to FVIII received BAY 94-9027 for 36 weeks on demand or prophylactically at intervals determined following a 10-week run-in period on 25 IU/kg body weight 2x/wk. Patients with >1 bleed during the run-in subsequently received 30-40 IU/kg 2x/wk; patients with ≤1 bleed were eligible for randomization to every-5-days (45-60 IU/kg) or every-7-days (60 IU/kg) prophylaxis (1:1) for 26 additional weeks until randomization arms were filled. Patients eligible but not randomized continued twice-weekly prophylaxis. Primary efficacy outcome was annualized bleeding rate (ABR).
The intent-to-treat population included 132 patients (prophylaxis, n=112; on demand, n=20). Median ABR (quartile [Q1;Q3]) for patients treated 2x/wk not eligible for randomization (n=13) improved after dose increase (17.4 [14.3;26.0] to 4.1 [2.0;10.6]). Median ABR for patients randomized to every-5-days treatment (n=43) was 1.9 (0;4.2), similar to patients eligible for randomization but who continued treatment 2x/wk (n=11). Median ABR for 32/43 patients (74%) who continued every-7-days prophylaxis until study end was 0.96 (0.0;4.3). 636/702 bleeds (90.6%) were controlled with ≤2 infusions. No patient developed a FVIII inhibitor.
BAY 94-9027 prevented bleeding across 3 individually tailored dose regimens and was effective for treatment of bleeds. This article is protected by copyright. All rights reserved.