Aspreva Pharmaceuticals Corp, of Victoria, BC, announced that the US FDA has granted Orphan Drug designation for mycophenolate mofetil (CellCept®; F. Hoffmann-La Roche) to treat pemphigus vulgaris (PV), a rare, severe chronic autoimmune disease that causes blisters of the skin and mucous membranes. Earlier in 2006, Aspreva announced the completion of patient enrollment in a global phase III clinical trial to evaluate mycophenolate mofetil in combination with corticosteroids in patients with active PV. The randomized, double-blind, placebo-controlled study will evaluate mycophenolate mofetil in 77 patients with active PV over a treatment period of 52 weeks. The primary endpoint encompasses both minimal disease activity, defined as no new persistent lesions, with a low steroid dose. Aspreva expects to complete the study in 2007.

CellCept is an immunosuppressant drug used in combination with other immunosuppressive drugs (eg, cyclosporine and corticosteroids) to prevent rejection in patients receiving heart, kidney, and liver transplants. In October 2003, Aspreva signed a collaboration agreement with Roche for the exclusive worldwide rights (excluding Japan) to develop and commercialize CellCept for all autoimmune disease applications. Aspreva has initiated two other global phase III clinical trials evaluating CellCept in active lupus nephritis and myasthenia gravis.

—A. Techman