Amicus starts trial for Fabry disease drug

The primary objective of the studies is to evaluate the safety and tolerability of treatment with Amigal, while the secondary objective is to evaluate certain pharmacodynamic measures of treatment.

An additional objective is the preliminary assessment of cardiac, renal and central nervous system function. The results of these clinical trials are expected to be available by the end of 2007.

Amigal is designed to selectively bind to and stabilize alpha-GAL, the enzyme deficient in Fabry disease.

Symptoms of the inheritable condition include pain, kidney failure and increased risk of heart attack and stroke. Fabry disease is estimated to affect approximately 5,000 to 10,000 people in the developed world, but recent evidence suggests that the disease may be significantly underdiagnosed.

The FDA has granted orphan designation for Amigal in the US, and the European Commission has designated Amigal as an orphan medicinal product in the EU.