Bayer Announces BAY94-9027 Biologics License Application Accepted by U.S. Food & Drug Administration

WHIPPANY, NJ, USA I October 30, 2017 I 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.

About Hemophilia A

Hemophilia has an estimated frequency of 1 in 5,000 male live births and affects approximately 400,000 people around the world, including an estimated 20,000 in the U.S. today.1 It is a largely inherited disorder in which one of the proteins needed to form blood clots is missing or reduced. In hemophilia A, the most common type of hemophilia, blood clotting is impaired as a result of a lack or defect of coagulation Factor VIII (FVIII). Patients therefore repeatedly experience bleeds in muscles, joints or other tissues, which can result in chronic joint damage. External injuries, even if they are trivial, can have serious consequences if not treated appropriately, as the blood clots more slowly than in healthy individuals.

Hemophilia treatment has advanced considerably over the past decades with life expectancy for people with hemophilia significantly increasing from about 11.4 years in 1920 to a potentially normal life span today.2 Today's research aims to reduce burden of treatment and improve the quality of life of people with hemophilia.

Bayer: Science For A Better Life

Bayer is a global enterprise with core competencies in the Life Science fields of health care and agriculture. Its products and services are designed to benefit people and improve their quality of life. At the same time, the Group aims to create value through innovation, growth and high earning power. Bayer is committed to the principles of sustainable development and to its social and ethical responsibilities as a corporate citizen. In fiscal 2016, the Group employed around 115,200 people and had sales of EUR 46.8 billion. Capital expenditures amounted to EUR 2.6 billion, R&D expenses to EUR 4.7 billion. These figures include those for the high-tech polymers business, which was floated on the stock market as an independent company named Covestro on October 6, 2015. For more information, go to www.bayer.us.

SOURCE: Bayer

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