AMT Obtains Orphan Drug Designation for Its LPL Gene Therapy Product

Mar 22 2004

AMT today announced that its adeno-associated viral vector (AAV) expressing lipoprotein lipase (LPL) product has been designated as an orphan medicinal product for the treatment of LPL deficiency by the European Commission.

This decision follows a positive recommendation by the European Agency for the Evaluation of Medicinal Products.

The designation confers regulatory support for accelerated development and ten years of market exclusivity after granting of marketing authorization. "LPL deficiency is an ideal target for gene therapy and our preclinical data give us great confidence that we may be able to rapidly provide a treatment in this area of unmet medical need," stated Dr. Jan Boesen, CEO of AMT. LPL deficiency is a disorder that affects a minimum of 5,000 patients in the Western world.

The disease is caused by a deficiency of LPL, the principal enzyme involved in the clearance of triglycerides from the plasma compartment. Humans who are deficient in LPL develop chronic pancreatitis, ultimately resulting in diabetes mellitus.

Currently, there is no specific treatment available to modulate the course of the disease other than a severe reduction of dietary fat. Gene therapy offers the prospect for effectively treating this disorder as well as potentially ameliorating the commonly associated diseases of coronary artery disease and myocardial ischemia.

AMT has recently announced that its AAV-mediated LPL gene therapy completely normalizes triglyceride levels in two different LPL deficient animal models. AMT anticipates treating the first patients early 2005.