Aspreva Pharmaceuticals Corporation and Roche have received an orphan drug designation for CellCept (mycophenolate mofetil) in the treatment of myasthenia gravis (MG) from the US Food and Drug Administration. Aspreva is currently evaluating CellCept for the treatment of myasthenia gravis in a global phase III study.

"We are pleased to have received this orphan drug designation for CellCept in the treatment of myasthenia gravis. This designation is an important step in our clinical development of CellCept for MG and in our efforts to deliver an effective, evidence-based treatment option for this debilitating and life-threatening illness," said Richard Glickman, Aspreva's chairman and chief executive officer.

The randomised, double-blind, placebo-controlled clinical trial now in progress is designed to evaluate the efficacy and safety of CellCept in maintaining or improving symptom control with reduced doses of corticosteroids in patients with myasthenia gravis over a treatment period of 36 weeks. The primary endpoints in the trial include both minimal disease activity and low steroid dose. Patient enrolment in the study is complete, and Aspreva expects to conclude the trial in late 2006, states a company release.

"Roche and Aspreva are highly committed to our partnership, to CellCept, and to developing better treatments for patients with a range of autoimmune diseases. With the potential to use CellCept in myasthenia gravis, we may be able to offer patients with few options a chance for better outcomes," said Dennis Burns, Roche's global head of business development.

The FDA's orphan drug designation is intended to encourage the development of new treatments for rare diseases. It is granted for treatments designed to prevent, diagnose or treat rare, life-threatening or chronically debilitating diseases that affect fewer than 200,000 people in the United States.

According to the Myasthenia Gravis Foundation, myasthenia gravis (MG) affects approximately 70,000 to 100,000 people worldwide, including approximately 36,000 people in the United States. MG is a debilitating, chronic autoimmune neuromuscular disease in which the body produces auto- antibodies which prevent the nerves from sending messages to the muscles.

CellCept is Roche's leading immunosuppressant or anti-rejection drug, used in combination with other immunosuppressive drugs (cyclosporine and corticosteroids) for the prevention of rejection in patients receiving heart, kidney and liver transplants.

Aspreva is an emerging pharmaceutical company focused on identifying, developing and, upon regulatory approval, commercializing new indications for approved drugs and late stage drug candidates for patients living with less common diseases.