Amicus Therapeutics will resume studies on a treatment for the genetic disorder Pompe disease in the next few months, the company said Tuesday.
The company said the Food and Drug Administration removed a clinical hold on testing of the therapy, and Amicus and regulators reached an agreement on the design of a new mid-stage trial. It plans to start the study in the first half of 2011 and report preliminary results in the second half of the year.
Amicus has been testing its drug AT2220 and enzyme replacement therapy as a treatment for Pompe disease. Patients with Pompe disease lack an enzyme in their blood that breaks down glycogen, a stored form of sugar used for energy. The glycogen collects in the muscles, heart, liver, and nervous system. That can cause muscle weakness and deadly respiratory problems.
Amicus placed testing of AT2220 on hold in February 2009, after two patients in a mid-stage clinical trial suffered serious side effects. The FDA then placed a clinical hold on the program, stopping any further human testing until the problem was resolved.
Shares of Amicus Therapeutics Inc. opened at $5.85 Tuesday, and have traded between $1.88 and $6.91 in the last year.