Bayer’s BAY94-9027 Biologics License Application accepted by U.S. FDA

The U.S. Food and Drug Administration (FDA) has accepted Bayer’s Biologics License Application (BLA) filing for BAY94-9027, an extended half-life site-specifically PEGylated recombinant human Factor VIII compound, for the treatment of hemophilia A in adults and adolescents 12 years of age and over. The regulatory submission is based on the results from the PROTECT VIII pivotal Phase II/III trial 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.  BAY94-9027 is an investigational agent and is not approved by the U.S. FDA, the European Medicines Agency (EMA) or other health authorities.

“The filing acceptance for BAY94-9027 by the FDA represents a milestone to Bayer in its commitment to seeking potential treatments to address the unmet needs of people living with hemophilia A,” said Dario Mirski, MD, Senior Vice President and Head of Medical Affairs Americas, Bayer. “For more than 25 years, Bayer has been committed to working with the community to support patients and deliver a spectrum of factor VIII products to provide physicians with treatment options for managing their patients with hemophilia A.”

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. An extended half-life recombinant factor VIII therapy may result in reduced frequency of infusions for people living with hemophilia A.

PROTECT VIII is a Phase II/III multicenter, multinational, partially randomized, open-label clinical trial with four treatment arms evaluating the safety and efficacy of BAY94-9027 in previously treated adults and adolescents with severe hemophilia A. In the three prophylactic treatment arms, efficacy and safety of BAY94-9027 was evaluated when used once every seven days, once every five days, or twice per week.

Earlier this fall, Bayer filed a Marketing Authorization Application (MAA) with the EMA. BAY94-9027 is an investigational agent and is not approved by the U.S. FDA, the EMA or other health authorities.