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FDA approves Nplate for long-term treatment of adult chronic ITP.

Amgen Inc. has announced that the FDA has approved Nplate(TM) (romiplostim), the first and only platelet producer for the treatment of thrombocytopenia in splenectomized (spleen removed) and non-splenectomized adults with chronic immune thrombocytopenic purpura (ITP). Nplate, the first FDA-approved peptibody protein, works by raising and sustaining platelet counts, representing a novel approach for the long-term treatment of this chronic disease.

Chronic ITP is a serious autoimmune disorder characterized by low platelet counts in the blood (thrombocytopenia), which can lead to serious bleeding events. Recognized as an orphan disease, chronic ITP affects an estimated 60,000 adult patients in the U.S. and is considered an unmet need by the FDA.

"Until now, patients suffering from chronic ITP have had limited available treatment options, many of which are often unsuitable for long-term use due to side effects and tolerability issues," said David J. Kuter, M.D., Chief of Hematology, Massachusetts General Hospital, Boston. "Nplate represents the first long-term treatment for adult chronic ITP patients, providing a new treatment approach for this chronic disease."

The FDA approval of Nplate was based on efficacy and safety results from two pivotal Phase 3 studies of adult patients with chronic ITP, including both splenectomized and non-splenectomized patients. The overall response rate for Nplate was 83 percent (n=69/83, p less than 0.0001) of treated splenectomized and non-splenectomized patients, and platelet counts were raised and sustained in these six month studies. Additionally, patients treated with Nplate were able to reduce or discontinue their use of concomitant ITP medications and emergency medications (i.e., corticosteroids, IVIG, Win-Rho, Anti-D therapy).

Specifically, in the Phase 3 studies, non-splenectomized patients had an 88 percent (n=36/41, p less than 0.0001) overall platelet response and splenectomized patients had a 79 percent (n=33/42, p less than 0.0001) overall platelet response rate. Combined data from both trials shows clinically relevant bleeding events were significantly reduced by half in patients treated with Nplate compared to placebo (15 percent vs. 34 percent, p=0.018). Amgen continues to study the long-term efficacy and safety of Nplate for which there is more than three years of follow up safety and efficacy data.

"For those suffering from ITP, the daily fear of experiencing a serious bleeding episode can be emotionally stressful and extremely difficult for both patients and their families. We welcome the addition of new treatment options which offer new hope for the treatment of this serious disease," said Craig Conway, executive director of the Platelet Disorder Support Association.
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Title Annotation:FDA Update: A monthly compendium of news features, announcements, regulatory issues and approvals from the Food & Drug Administration.
Publication:Pharmaceutical Processing
Date:Sep 1, 2008
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