Aspreva, Roche win orphan status for CellCept to treat myasthenia gravis

By E. Janene Geiss

Philadelphia, Jan. 3 - Aspreva Pharmaceuticals Corp. and Roche announced Tuesday that the U.S. Food and Drug Administration has granted an orphan drug designation for CellCept (mycophenolate mofetil) in the treatment of myasthenia gravis.

Aspreva is currently evaluating CellCept for the treatment of myasthenia gravis in a global phase 3 study, according to a company news release.

"We are pleased to have received this orphan drug designation for CellCept in the treatment of myasthenia gravis," Richard Glickman, Aspreva's chairman and chief executive officer, said in the release.

The randomized, 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 enrollment in the study is complete and Aspreva said it expects to conclude the trial in late 2006.

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 affects about 70,000 to 100,000 people worldwide, including about 36,000 people in the United States. It is a debilitating, chronic autoimmune neuromuscular disease in which the body produces auto-antibodies that prevent the nerves from sending messages to the muscles. Although the disease can affect any voluntary muscle, it frequently involves those controlling eye movements, chewing, swallowing, coughing and facial expressions, but can be more severe in some patients.

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.

In October 2003, Aspreva announced a collaboration agreement with Roche for the exclusive worldwide rights (excluding Japan) to develop and, upon regulatory approval, commercialize CellCept for all autoimmune disease applications.

CellCept is not currently approved for the treatment of any autoimmune disease, including myasthenia gravis.

Aspreva is a Victoria, B.C., pharmaceutical company focused on identifying, developing and commercializing new indications for approved drugs and late-stage drug candidates for patients living with less common diseases.

Roche, based in Basel, Switzerland, is a research-driven health care company focused on pharmaceuticals and diagnostics.

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