FDA Approves Amgen’s Nplate

The FDA has approved Amgen‘s Nplate, 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.

The FDA approval of Nplate was based on efficacy and safety results from two Phase III studies of adult patients with chronic ITP, including both splenectomized and non-splenectomized patients. The overall response rate for Nplate was 83% of treated 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.

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.”

Said Roger M. Perlmutter, M.D., Ph.D., Amgen’s executive vice president of R&D, “The FDA approval of Nplate is the result of more than 15 years of research and represents an important biotechnology milestone as it is the first FDA-approved peptibody protein, an innovative platform for delivering targeted therapies.”

Nplate was also approved for ITP by Australia’s Therapeutic Goods Administration (TGA) in July 2008. Amgen has filed for regulatory approval of Nplate in the European Union (EU), Canada, and Switzerland and these applications are currently under review. Nplate has also received orphan designation for ITP in the EU (2005), Switzerland (2005) and Japan (2006).

Amgen is continuing 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.