FDA Accepts Bayer BLA for Hemophilia A Therapy

FDA has accepted a Bayer BLA for BAY94-9027, an extended half-life PEGylated recombinant human Factor VIII compound for treating hemophilia A in adults and adolescents 12 years of age and older. The submission is based on data from the PROTECT VIII pivotal Phase 2-3 trial that was designed to evaluate the efficacy and safety of BAY94-9027 when used on-demand and prophylactically once every seven days, once every five days, or twice per week, according to the company.

“BAY94-9027 is engineered to potentially prolong FVIII activity in the blood while preserving coagulation activity using site-specific PEGylation technology, where a PEG (Polyethylene glycol) molecule is consistently attached to the factor VIII protein at a specific site,” the company says. “An extended half-life recombinant factor VIII therapy may result in reduced frequency of infusions for people living with hemophilia A.”