Enzyme Therapy Slows Kidney Function Decline, Study Suggests

The researchers pooled the results of three previous clinical trials of ERT with agalsidase alfa in 108 men with Fabry disease—a rare genetic disorder. Without treatment, Fabry disease causes progressive loss of kidney function, eventually leading to end-stage renal disease.

During treatment with an inactive placebo, kidney function declined rapidly. By comparison, during treatment with agalsidase alfa (1 to 4.5 years), the rate of decline slowed considerably. The response to treatment was not as good for patients with lower initial kidney function. "This underlines the importance of prompt diagnosis and intervention in patients with Fabry disease," adds Dr. West.

The study was one of the largest ever of men with Fabry disease and included the most accurate techniques of measuring kidney function. The study also had important limitations: it used data from different studies performed at different times; it represented a relatively narrow range of patient characteristics, specifically excluding children and women; and it lacked adequate data for full statistical analysis.

While not approved for use in the US, Agalsidase alfa is approved for use in over 40 countries including Canada, European countries, Argentina, Australia, and Japan. A related drug called agalsidase beta is approved in the US.

Dr. West has received research funding and other fees from Shire Human Genetic Therapies Inc. and other drug companies.