FDA Accepts Bayer's Biologics License Application for Investigational Treatment Option in Hemophilia A

WHIPPANY, N.J., March 4, 2015 /PRNewswire/ -- Bayer HealthCare today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's Biologics License Application (BLA) for BAY 81-8973, a recombinant Factor VIII compound.  Bayer is seeking FDA approval of the investigational compound, proposed trade name Kovaltry™, for the treatment of hemophilia A in children and adults.

"Bayer is committed to continually bringing new therapies to hemophilia A patients who need them," said David Weinreich, M.D., Head, Global Development, Specialty Medicine, Bayer HealthCare Pharmaceuticals.  "Today's milestone represents the next step in that commitment."

The BLA is based on results from the LEOPOLD (Long-Term Efficacy Open-Label Program in Severe Hemophilia A Disease) clinical trials, which evaluated BAY 81-8973 in adults and children using two- and three-times-per-week prophylaxis dosing regimens.  The LEOPOLD Clinical Development Program consists of three multinational clinical trials designed to evaluate the pharmacokinetics, efficacy, and safety of BAY 81-8973 in subjects with severe hemophilia A (<1% FVIII:C).

BAY 81-8973 is an investigational agent and is not approved by the United States FDA, the European Medicines Agency (EMA) or other health authorities.

Hemophilia A, also known as factor VIII deficiency or classic hemophilia, is a largely inherited bleeding disorder in which one of the proteins needed to form blood clots in the body is missing or reduced. Hemophilia A is the most common type of hemophilia and is characterized by prolonged or spontaneous bleeding, especially into the joints, muscles or internal organs.  It is estimated that 20,000 people are living with hemophilia in the U.S. today.ⁱ

Bayer HealthCare is committed to delivering science for a better life by advancing a portfolio of treatments. Hematology at Bayer HealthCare includes numerous compounds in various stages of development for hemophilia, sickle cell anemia, and other blood and bleeding disorders. Together, these compounds reflect the company's commitment to research and development, prioritizing specific targets for intervention with the potential to improve the way that rare blood and bleeding disorders are treated.

About Bayer HealthCare Pharmaceuticals Inc.
Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world's leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Medical Care, and Pharmaceuticals divisions. As a specialty pharmaceutical company, Bayer HealthCare provides products for General Medicine, Hematology, Neurology, Oncology and Women's Healthcare. The company's aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases.

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Forward-Looking Statements
This news release may contain forward-looking statements based on current assumptions and forecasts made by Bayer Group or subgroup management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer's public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

ⁱ http://www.cdc.gov/ncbddd/hemophilia/data.html

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SOURCE Bayer HealthCare