Enzyme Replacement Therapy Effective And Safe For Women.(Brief Article)

2001 OCT 11 - (NewsRx.com & NewsRx.net) --

Transkaryotic Therapies, Inc. announced at a workshop held in conjunction with the 39th Annual Symposium of the Society for the Study of Inborn Errors of Metabolism in Prague, Czech Republic, September 6, 2001, new data about Replagal (agalsidase alfa) enzyme replacement therapy from the first ever study of females with Fabry disease.

In addition, leading European clinicians made a series of presentations on new data relating to the long-term safety and efficacy of Replagal.

In a 15 patient study of females with Fabry disease, Drs. Michael Beck, Frank Bahner, and Christoph Kampmann of The University Hospital in Mainz, Germany reported that the pharmacokinetics of Replagal in both males and females were similar, suggesting that delivery of Replagal to tissues in women is similar to that in men. In addition, the investigators reported substantial cardiac improvement in these patients and concluded that Replagal therapy could have an important clinical benefit for women. Beck and his colleagues presented data suggesting that essentially all females with Fabry disease are symptomatic.

In a separate open-label extension study with male patients receiving Replagal for two years, Thomas J. Schuetz, MD, PhD, TKT, reported that Replagal has been well-tolerated and no patient has experienced an infusion reaction after six months of treatment. All patients on Replagal therapy in this study are now on home therapy, with most receiving the drug at home for over one year. In addition, he reported that approximately 200 patients have received Replagal therapy worldwide, and less than 10% have experienced minor, generally self-resolving, infusion reactions.

"We are excited by the reports from both of these studies," noted Rolf Gunnarsson, MD, PhD, TKT Europe-5S AB, organizer of the workshop. "Many clinicians wrongly assume that women do not suffer from this painful, life-threatening disease. This study will help counteract this impression. Furthermore, we are pleased by the ...