FDAnews Drug Daily Bulletin
Pharmaceuticals / Research and Development

Bayer: Recombinant Factor VIII Proves Effective Prophylaxis in Phase III Hemophilia A Trial

Feb. 19, 2014

Bayer announced Tuesday that its BAY94-9027 recombinant factor VIII met its primary efficacy endpoint of protection from bleeds with fewer infusions in a pivotal Phase II/III trial.

PROTECT VIII is a multicenter, multinational, partially randomized and open-label study of 134 previously treated adults and adolescents with severe hemophilia A. Forty-four percent of patients in the five-day treatment arm experienced no bleeds, as did 37 percent of subjects receiving an infusion every seven days, Bayer said. The 13 subjects in the twice-per-week treatment arm, who experienced higher bleeding rates during the assessment period, reduced their median annualized bleeding rate from 17.4 to 4.1 following dose increase, the company added.

Currently marketed factor VIII infusions have a short half-life, requiring infusion as often as every two days, according to Bayer. The German drugmaker extends the BAY94-9027’s half-life while preserving full biologic activity by inserting a single cysteine, an amino acid, onto the factor VIII surface.

The drug also met its safety endpoints, with patients tolerating it and developing no inhibitors during the 36 weeks of treatment. Bayer plans to study the drug’s use during major surgery, in children and in previously untreated patients. —Lena Freund

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