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FDA Approves Prophylactic Use of Kogenate® FS

The U.S. Food and Drug Administration (FDA) approved a new use for Kogenate® FS Antihemophilic Factor (Recombinant), a fVIII drug manufactured by Bayer HealthCare LLC. While Kogenate® FS had already been approved for the control and prevention of bleeding episodes and peri-operative management in adults and children (0-16 years) with hemophilia A, this additional indication was made to reduce the frequency of bleeding episodes and prevent joint damage in children with severe hemophilia A.

The new approval represents a prophylactic (preventive) approach to hemophilia treatment. Regular weekly infusions help prevent repeated joint bleeds that can cause chronic pain, impaired mobility and arthritis. Prophylaxis has also been recommended by the National Hemophilia Foundation’s Medical and Scientific Advisory Council (MASAC) in its Recommendation #179 “Concerning Prophylaxis (Regular Administration of Clotting Factor Concentrate to Prevent Bleeding).” The council considers it an optimal therapy for young children with severe hemophilia A and B.

“Administering Kogenate FS to children with hemophilia A on a daily basis before a bleeding event occurs will reduce bleeding into joints and help prevent joint damage, a major cause of disability in hemophiliacs,” said Jesse Goodman, MD, MPH., director, FDA’s Center for Biologics Evaluation and Research.

The seminal research and impetus for the new approval originated from the Joint Outcomes Study, a multi-center trial designed to compare on-demand vs. prophylaxis treatment approaches and their effect on joint health. The study, completed in 2005, was led by Marilyn J. Manco-Johnson, MD, Professor of Pediatrics and Associate Professor of Pathology in the Department of Pediatrics, University of Colorado at Denver and Health Sciences Center and Director of the Mountain States Regional Hemophilia & Thrombosis Center at the University of Colorado.

Study participants included 65 boys with factor VIII deficiency who were between one and two-and-a-half years old. The study measured key results, such as frequency of joint hemorrhages and level of joint function, for up to 5.5 years. While 32 of the prophylaxis patients were given regular, every-other-day infusions of Kogenate FS, 33 were treated intensively with multiple infusions of the product but only at the onset of a joint hemorrhage. 

The boys participated in the study until they reached 6 years old. Their joint structure and function were then measured using X-rays, magnetic resonance imaging (MRI) and physical exams. Results indicated that 93% of children in the prophylaxis group showed normal joints, in contrast to 55% in the on-demand group. The trial results demonstrated that an early, every-other-day treatment regimen improved joint function in contrast to an aggressive, on-demand approach. Overall, there was an 83% reduction in the risk for joint damage in patients receiving prophylaxis from an early age.

“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,” said Craig Kessler, MD, Georgetown University Hospital and Chair of NHF’s MASAC. “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.”

On a related note, the European Medicines Agency, the European equivalent of the FDA, has granted license approval to Bayer HealthCare LLC for its new sterile filling facility in Berkeley, CA. The facility will be employed in the late-stage production processes of filling and lyophilization (freeze-drying) for Kogenate® FS, both for US and European markets. The company cited several advantages of the facility including increased automation to minimize risk of external contamination, greater assurance of product quality, improved process reliability and expanded capacity for future hemophilia therapies.

Source: Bayer HealthCare news releases dated October 10 and 27, 2008; FDA news release dated October 10, 2008