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news.Genzyme, a biotechnology company, has started enrollment in a world-wide post-marketing clinical trial evaluating the efficacy and safety of treating pediatric male patients with mild Fabry disease symptoms with a low-dose regimen of Fabrazyme.
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Approximately 20 institutions across Europe, the US, Canada and Latin America are expected to participate in the study. The trial will examine the efficacy and safety of two lower-dose regimens of Fabrazyme in male patients aged five to 18 years over the course of five years.
Patients will receive either half the recommended dose of Fabrazyme every two weeks (0.5mg/kg of body weight) or a dose of 1mg/kg every four weeks. Fabrazyme at the recommended dose of 1mg/kg every two weeks has been approved in more than 40 countries, including the US and Canada, as well as throughout the EU, for the treatment of Fabry disease.
According to the company, the data from the ‘Fabrazyme: intervening early at a lower dose (FIELD)’ study may support supplemental submissions to regulatory agencies seeking additional dosing options that facilitate early treatment for Fabry disease.
Uma Ramaswami, co-principal investigator for the FIELD study, said: “This treatment regimen might be more adapted for those patients with milder symptoms of the disease, providing them flexibility in their treatment options. The ability to provide a lower or less frequent dose of Fabrazyme for pediatric patients with milder symptoms may translate to some patients receiving the treatment early.”