Bayer HealthCare LLC announced the U.S. Food and Drug Administration has approved routine prophylaxis with Kogenate

Bayer HealthCare LLC announced today that the U.S. Food and Drug Administration (FDA) has approved routine prophylaxis with Kogenate® FS Antihemophilic Factor (Recombinant) to reduce the frequency of bleeding episodes and the risk of joint damage in patients aged 0-16 years with severe hemophilia A with no pre-existing joint damage. This important approval provides these patients with the only factor VIII treatment that the FDA has determined safe and effective for routine prophylaxis -- a treatment regimen recommended by the National Hemophilia Foundation's Medical and Scientific Advisory Council (MASAC).

"The FDA approval of Kogenate FS as the first factor VIII treatment product in the U.S. to be used to replenish factor VIII levels in a prophylactic manner marks a significant milestone in the care of patients, especially young children, with hemophilia A," said Craig Kessler, M.D., Georgetown University Hospital and Chair, MASAC. "The results from the pivotal clinical study confirmed that the administration of Kogenate FS to prevent bleeding into the joints was more beneficial to joint health and function than 'on-demand' treatment of acute episodes of joint bleeds."

Dr. Kessler added, "The data justify the consideration of prophylaxis treatment for children with severe and moderate severity hemophilia A, uncomplicated by pre-existing joint damage, to be the medical standard of care. The FDA's recognition that Kogenate FS is an effective prophylactic FVIII replacement product has 'jump started' the standard of hemophilia care in the United States so that it is now on par with other developed countries, especially those in Western and Northern Europe."

The FDA approval of Kogenate® FS for routine prophylaxis in children without pre-existing joint damage is based on the clinical data from a multicenter trial in the U.S. that included 65 boys with severe hemophilia A less than 30 months of age at study entry. Study participants were followed for up to 5.5 years. This Joint Outcomes Study (JOS), conducted over a 10-year period, was led by Marilyn J. Manco-Johnson, M.D., Professor of Pediatrics and Associate Professor of Pathology in the Department of Pediatrics, University of Colorado at Denver and Health Science Center, and Director of the Mountain States Regional Hemophilia & Thrombosis Center at the University of Colorado. Key findings from the JOS study, published in the August 9, 2007 issue of The New England Journal of Medicine(2), include:

* 93 percent of the participants in the routine prophylaxis group showed normal joint function, in contrast to 55 percent in the episodic group.
* Kogenate FS prophylaxis treatment was able to preserve the joint even if the child had less than or equal to two bleeds per index joint.
* Patients from the prophylaxis group were eight times more likely to have damage-free joints than those from the episodic group.
* The prophylaxis group had an 81.5 percent reduction in annual bleeding frequency compared to the episodic group.
* Overall, there was an 83 percent reduction in the risk for joint damage in patients receiving prophylaxis from an early age.
* Ten percent of the patients treated episodically experienced life-threatening bleeds compared to zero patients treated prophylactically.
* The most common adverse events were related to central venous access, such as catheterization and catheter removal, central line infection and pyrexia.

The study was designed with special emphasis on the "index joints," including the elbow, knee and ankle joints, which are most prone to bleeding in severe hemophilia patients. Joint structural outcomes and functioning were measured at 6 years of age by radiography, magnetic resonance imaging (MRI) and physical exams.

"The value for prophylaxis for hemophilia A patients has been understood for many years, but until today we have not had the combination of clinical evidence, pharmaceutical indication and the alignment of healthcare professionals, patients and regulators needed to ensure this treatment option is available," said Val Bias, CEO, National Hemophilia Foundation. "Bayer HealthCare's pursuit of this approval shows leadership and commitment to providing the scientific evidence that proves the benefits of prophylactic use for joint health in pediatric hemophilia patients."

This U.S. FDA approval may positively affect the prophylactic use of Kogenate FS in certain developing markets, including countries in Asia and South and Central America, where the product is approved based on the U.S. label.

"Today's announcement is a milestone in Bayer HealthCare's continuing commitment to advancing the science and treatment for the hemophilia community," said Paul Bedard, Vice President, General Manager, Hematology Business Unit, Bayer HealthCare. "From the beginning, our goal in pursuing this indication was to provide treatment options that would reduce bleeding episodes and protect the joint health of children with hemophilia A, which are the everyday concerns of patients."

About Kogenate® FS

Kogenate® FS, Antihemophilic Factor (Recombinant), is a recombinant factor VIII treatment indicated for the control and prevention of bleeding episodes and peri-operative management in adults and children (0-16 years) with hemophilia A. Kogenate FS is also indicated for routine prophylaxis to reduce the frequency of bleeding episodes and the risk of joint damage in children with hemophilia A with no pre-existing joint damage. The most serious adverse reactions are systemic hypersensitivity reactions including bronchospastic reactions and/or hypotension and anaphylaxis and the development of high-titer inhibitors necessitating alternative treatments to AHF. The most common adverse reactions observed in clinical trials (frequency greater than or equal to 4% of patients) were skin-associated hypersensitivity reactions (rash, pruritus, urticaria), inhibitor formation in previously untreated or minimally treated patients, infusion site reactions, and central venous access device (CVAD) line-associated infections.

Kogenate FS is contraindicated in patients who have manifested life-threatening immediate hypersensitivity reactions, including anaphylaxis, to the product or its components, including mouse or hamster proteins.

Please see the full prescribing information for important risk and use information at www.kogenatefs.com.