FDA Approves Antihemophilic Factor for Routine Prophylaxis in Children With Hemophilia A

Yael Waknine

August 18, 2009

August 18, 2009 — The US Food and Drug Administration (FDA) has approved a new indication for antihemophilic factor (recombinant) intravenous injection (Helixate FS, CSL Behring) for routine prophylaxis to reduce the frequency of bleeding episodes and the risk for joint damage in children aged 16 years or younger with hemophilia A and no preexisting joint damage. The recommended dose is 25 IU/kg administered every other day.

"Bleeding into joints, including the ankles, elbows and knees, is a concern for children with hemophilia A and their treatment providers," said Jerry S. Powell, MD, director of the Hemophilia Treatment Center at the University of California–Davis, in a company news release. "The FDA's approval of Helixate FS for routine prophylaxis is an innovation that will help our patient population effectively manage their condition. Modern treatment of hemophilia means preventing bleeding through routine prophylaxis, and we eagerly anticipate our patients reaching young adulthood with minimal joint damage and the ability to pursue normal lives."

The approval was based on data from a multicenter, open-label, prospective randomized study of 65 boys younger than 30 months, showing that prophylactic use of antihemophilic factor yielded less joint damage than episodic treatment, as detected by magnetic resonance imaging or radiography (7% vs 42%; P = .002). The mean annual frequency of bleeding episodes was likewise reduced (index joint hemorrhages, 0.63 bleeds/year vs 4.89 bleeds/year).

On a per joint basis, joints in the regular prophylaxis arm were 8-fold more likely to remain damage-free than those in the episodic arm. Joint damage was most frequently observed in ankle joints; ankles were also the index joint that demonstrated the highest frequency of bleeding events in this study.

The study data also demonstrated that antihemophilic factor is safe and effective for routine use. Adverse events most commonly reported with product use are inhibitor formation in previously untreated or minimally treated patients, skin-associated hypersensitivity reactions, infusion site reactions, and central venous access device line-associated infections. Serious adverse events may include systemic hypersensitivity reactions and the development of high-titer inhibitors necessitating alternative treatments to antihemophilic factor.

Antihemophilic factor intravenous injection previously was approved for the control and prevention of bleeding episodes in hemophilia A in patients aged 0 to 16 years.

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