Genzyme Corporation has got the thumbs up from Europe’s scientific advisory body, the CHMP, to include new evidence of efficacy in its labelling for the Fabry disease treatment Fabrazyme (agalsidase beta) following positive data from the world’s largest ever study of the condition.
The company will now be able to include the following information in its product information: that “the rate of clinical events was substantially lower among Fabrazyme-treated patients compared to placebo-treated patients;” and that “Fabrazyme treatment at 1 mg/kg every other week provides clinical benefit on key clinical outcomes in patients with early and advanced Fabry disease.” Dr Stephen Waldek, principal investigator of the study, from Hope Hospital in Manchester, UK, said: “This trial provides the first randomised, placebo-controlled evidence that enzyme replacement therapy impacts the clinical outcomes that cause morbidity and mortality in Fabry disease.”
The CHMP’s recommendation will now be forwarded to the European Commission for final approval in all EU member states, Iceland and Norway. These data are also currently under review by the US Food and Drug Administration.
Fabrazyme was first backed in the European Union in 2001, and given a nod of approval in the USA two years later [[16/01/03b]], [[25/04/03b]]. Fabry disease is a condition whereby lipids accumulate within the kidneys, heart and other organs, leading to kidney failure, stroke and heart disease. The average life expectancy of patients with the disease is 43 years.