Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.The FDA has granted orphan drug designation for Aspreva Pharmaceuticals and Roche's CellCept for its potential use as a treatment for the debilitating neuromuscular disease myasthenia gravis.
Subscribe to our email newsletter
Aspreva is currently conducting a global phase III study evaluating the efficacy and safety of CellCept in maintaining or improving symptom control with reduced doses of corticosteroids in patients with myasthenia gravis (MG) 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.
“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 US.