Nplate—Top Drug Innovation for 2008?

Fred Cohen, M.D., editor of the Pharma’s Cutting Edge blog, considers both Nplate and its fellow thrombopoeitin receptor Promacta to be among the top drug innovations for 2008. Why?

“(The TPO-agonist class) represents a potential major departure in the treatment of this uncommon, costly disease.”

Patients suffering from chronic ITP, or immune thrombocytopenic purpara, suffer platelet destruction and are unable to produce enough new platelets to compensate. Ecessive bleeding may result. The condition can often resolve itself, particularly in children, and is sometimes mild enough to require monitoring rather than treatment. Chronic sufferers, however, have a tougher row to hoe. Roughly 140,000 patients are currently being treated for chronic ITP in the United States and Europe.

The treatment goal is to maintain enough platelets to avoid bleeding events.

Results from an ongoing open-label extension study were recently reported at the 2008American Society of Hematology meeting in San Francisco, California. The long-term interim data shows that treatment with Nplate, which is approved for adults that have not responded to other forms of treatment such as corticosteroids, maintains platelet levels for up to four years.

The most commonly reported side-effects included headaches (34%) and confusion (30%), fatigue (30%), and nasopharyngitis (30%).

Follow-up on Nplate’s long-tem effectiveness and safety is continuing. At this point, only some 20 patients have been followed for more than three years.

Do you suffer from chronic ITP? How are you treating it? And if you’re being treated with Nplate, how is it working for you?