NplateTreatment for Idiopathic (Immune) Thrombocytopenic Purpura
Update: Nplate Now FDA Approved - August 22, 2008
Amgen to Discuss Romiplostim BLA
Amgen to Discuss Romiplostim Application at FDA ODAC Meeting
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Mar 12, 2008 - Amgen Inc. will discuss the proposed Biologic License Application (BLA) for romiplostim, an investigational thrombopoietin mimetic peptibody, for the treatment of thrombocytopenia in adult patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) today at the Oncologic Drugs Advisory Committee (ODAC). Patients with chronic ITP, a serious autoimmune disorder characterized by low platelet counts in the blood (thrombocytopenia) face the risk of bleeding events. Treatment of thrombocytopenia in adult patients with chronic ITP is considered an unmet medical need by the U.S. Food and Drug Administration (FDA) and has received orphan drug designation.
The proposed indication for romiplostim submitted to the FDA is for the treatment of thrombocytopenia in adults with chronic ITP who have not undergone splenectomy (removal of the spleen) and have had an inadequate response or are intolerant to corticosteroids and/or immunoglobulins; or patients who have had their spleen removed and have an inadequate response to the procedure.
Amgen's Commitment to Risk Management
Amgen is committed to a robust risk management program for romiplostim. The proposed comprehensive risk management program includes additional clinical studies and a variety of measures designed to ensure appropriate use of romiplostim in ITP patients.
"By stimulating platelet production, romiplostim represents a potentially new approach to the management of chronic ITP," said David J. Kuter, M.D., D. Phil., Chief of Hematology, Massachusetts General Hospital, Boston. "Romiplostim would be an important treatment option for adult patients affected by this disease."
Amgen filed for regulatory approval of romiplostim with the FDA in October 2007 and was granted priority review. Regulatory filings in the European Union (EU), Canada and Australia were also filed in 2007 and are currently under review. Orphan designation was granted for romiplostim in 2003 by the FDA. An orphan disease is defined as a condition that affects fewer than 200,000 people nationwide. Romiplostim also has received orphan designation for the proposed indication in the EU (2005), Switzerland (2005) and Japan (2006).
Romiplostim is an investigational protein ("peptibody"), containing two components--a "binding" peptide linked to a larger protein. Developed by Amgen, peptibodies are engineered therapeutic molecules that can bind to human drug targets and contain peptides linked to the constant domains of antibodies. Romiplostim works similarly to thrombopoietin (TPO), a natural protein in the body. The binding peptide component of romiplostim stimulates the TPO receptor, which is necessary for growth and maturation of bone marrow cells that produce platelets.
About Adult ITP
Platelets are blood cells needed to prevent bleeding. Low platelet counts leave adult ITP patients open to sudden serious bleeding events, making it impossible to arrest blood flow. The risk for serious bleeding events increases when platelet counts drop to less than 30,000 platelets per microliter.
There are limited FDA approved treatments (i.e., corticosteroids, immunglobulins) or surgical therapy (removal of the spleen) for adult patients with chronic ITP. There are an estimated 60,000 adult patients with chronic ITP in the United States. ITP affects about twice as many adult women as men.
With ITP, platelets are destroyed by the patient's own immune system. ITP has historically been considered a disease of platelet destruction. However, recent data also suggest that the body's natural platelet production processes are unable to compensate for low levels of platelets in the blood. Increasing the rate of platelet production may address low platelet levels associated with ITP.
Amgen discovers, develops, manufactures and delivers innovative human therapeutics. A biotechnology pioneer since 1980, Amgen was one of the first companies to realize the new science's promise by bringing safe and effective medicines from lab, to manufacturing plant, to patient. Amgen therapeutics have changed the practice of medicine, helping millions of people around the world in the fight against cancer, kidney disorder, rheumatoid arthritis, and other serious illnesses. With a deep and broad pipeline of potential new medicines, Amgen remains committed to advancing science to dramatically improve people's lives. To learn more about our pioneering science and our vital medicines, visit www.amgen.com.
This news release contains forward-looking statements that are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and assumptions that could cause actual results to differ materially from those described. All statements, other than statements of historical fact, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial metrics, expected legal, arbitration, political, regulatory or clinical results or practices, customer and prescriber patterns or practices, reimbursement activities and outcomes and other such estimates and results. Forward-looking statements involve significant risks and uncertainties, including those discussed below and more fully described in the Securities and Exchange Commission (SEC) reports filed by Amgen, including Amgen's most recent annual report on Form 10-K and most recent periodic reports on Form 10-Q and Form 8-K. Please refer to Amgen's most recent Forms 10-K, 10-Q and 8-K for additional information on the uncertainties and risk factors related to our business. Unless otherwise noted, Amgen is providing this information as of March 12, 2008 and expressly disclaims any duty to update information contained in this news release.
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Amgen, Thousand Oaks
Christine Regan, 617-359-1324 (media)
John Shutter, 805-447-1060 (investors)
Posted: March 2008
- Nplate (romiplostim) Now Approved For Earlier Use In Adults With Immune Thrombocytopenia - October 18, 2019
- FDA Approves Nplate (Romiplostim) For Use In Pediatric Patients With Immune Thrombocytopenia - December 14, 2018
- FDA Approves Nplate for Long-Term Treatment of Adult Chronic ITP - August 22, 2008
- Amgen's FDA Nplate Approval Statement Retracted - July 15, 2008
- FDA Approves Nplate for Long-Term Treatment of Adult Chronic ITP - July 14, 2008
- Amgen Statement on Successful Outcome of Romiplostim Panel Meeting - March 13, 2008
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