�Amgen Inc. (NASDAQ: AMGN) announced that the United States (U.S.) Food and Drug Administration (FDA) has approved Nplate� (romiplostim), the first and only platelet producer for the handling of thrombopenia in splenectomized (spleen removed) and non-splenectomized adults with chronic immune thrombocytopenic purpura (ITP). Nplate, the first base FDA-approved peptibody protein, works by raising and sustaining platelet counts, representing a novel approaching for the long-term intervention of this chronic disease.
Chronic ITP is a serious autoimmune disorder characterized by low platelet counts in the blood (thrombocytopenia), which can buoy lead to serious bleeding events. Recognized as an orphan disease, chronic ITP affects an estimated 60,000 grownup patients in the U.S. and is considered an unmet demand by the FDA.
"Until now, patients suffering from chronic ITP have had limited available treatment options, many of which are often unsuitable for long-run use referable to slope effects and tolerability issues," said David J. Kuter, M.D., Chief of Hematology, Massachusetts General Hospital, Boston. "Nplate represents the first long-term handling 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 deuce 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
Specifically, in the Phase 3 studies, non-splenectomized patients had an 88 pct (n=36/41, p
"For those excruciation from ITP, the daily fear of experiencing a serious hemorrhage episode stool be emotionally stressful and extremely difficult for both patients and their families. We welcome the gain of new treatment options which offer new hope for the treatment of this life-threatening disease," aforementioned Craig Conway, executive director of the Platelet Disorder Support Association.
In accession to improved clinical benefits, described in the FDA labeling, Amgen believes Nplate offers patients a confirming net health benefit with fewer hospitalizations from bleeding events, as well as reduced motivation for emergency medications (IVIG and Win-Rho). Amgen expects the tote up costs of care for chronic ITP patients managed with Nplate to be less than or comparable to the total costs of tutelage with banner treatment regimens.
Amgen besides announced it will set in motion the Nplate� NEXUS (Network of EXperts Understanding and Supporting Nplate� and Patients) Program, a multi-faceted political program designed to provide comprehensive access, reinforcement and education for inveterate ITP patients, their caregivers and health care providers. The Nplate� NEXUS Program is part of the Risk Evaluation and Mitigation Strategy (REMS) developed by Amgen in partnership with the FDA to assure safe use of Nplate patch minimizing peril. The programme will help appropriate use of Nplate, provide patient support through education and resources and help with ongoing surveil up through safety data collection.
Through the Nplate� NEXUS Program, eligible patients who ar uninsured, underinsured, or ineffective to afford their insurance co-payments may be able to pick up reimbursement documentation and other assistance from Amgen. For example, ane such programme helps cover up to 50 percent of an eligible, commercially-insured patient's co-payments for Nplate. Recognizing that some patients may not have health care coverage, Amgen continues to offer another program for all of its innovative products, including Nplate, which provides product free of charge to eligible, low-income patients without insurance.
"Amgen is committed to forward-moving the discovery and exploitation of new therapies for grievous illnesses where there is unmet medical need," said Roger M. Perlmutter, M.D., Ph.D., executive frailty president of Research and Development at Amgen. "The FDA approval of Nplate is the result of more than 15 years of research and represents an of import biotechnology milepost as it is the first FDA-approved peptibody protein, an forward-looking platform for delivering targeted therapies."
Nplate was too approved for ITP by Australia's Therapeutic Goods Administration (TGA) in July 2008. Amgen has filed for regulatory blessing 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).
More information about the Nplate� NEXUS Program is available by calling 1-877-NPLATE1 (1-877-675-2831), or by visiting http://www.nplate.com.
About Adult ITP
Platelets are blood cells requisite to forestall 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 thrombocyte counts drop to less than 30,000 platelets per microliter.
There are limited approved treatments (i.e., corticosteroids, immunoglobulins) or surgical therapy (removal of the spleen) available to adult patients with chronic ITP. Currently, there ar 140,000 treated chronic ITP patients in the U.S. and Europe. ITP affects around twice as many adult women as men.
With ITP, platelets are destroyed by the patient's possess immune system. ITP has historically been considered a disease of platelet destruction. However, recent data too suggest that the body's natural thrombocyte production processes are unable to indemnify for low levels of platelets in the lineage. Increasing the rate of platelet production may address low thrombocyte levels associated with ITP.
About Nplate
Nplate, Amgen's first peptibody protein, is a novel engineered therapeutical fusion protein with attributes of both peptides and antibodies, simply is discrete from each. Nplate works similarly to thrombopoietin (TPO), a instinctive protein in the body. Nplate stimulates the TPO receptor, which is necessary for growth and ripening of bone marrow cells that produce platelets.
Important Safety Information
Serious untoward reactions associated with Nplate in clinical studies were bone marrow reticulin deposition and deterioration thrombocytopenia later Nplate discontinuation.
Bone Marrow Reticulin Formation and Risk for Bone Marrow Fibrosis
- Nplate presidential term increases the risk for development or progression of reticulin fiber deposition inside the off-white marrow.
- In clinical studies, Nplate was discontinued in four of the 271 patients because of pearl marrow reticulin deposition. Six additional patients had reticulin observed upon bone centre biopsy. All 10 patients with bone marrow reticulin deposition had received Nplate doses ? 5 mcg/kg, and 6 received doses ? 10 mcg/kg.
- Progression to marrow fibrosis with cytopenias was not reported in the controlled clinical studies. In the extension study, one patient with ITP and hemolytic genus Anemia developed marrow fibrosis with collagen during Nplate therapy.
- Clinical studies have non excluded a risk of bone marrow fibrosis with cytopenias.
- Prior to knowledgeability of Nplate examine the peripheral blood smear closely to establish a service line level of cellular structural abnormalities. Following identification of a stable Nplate cupid's disease, examine peripheral blood smears and CBCs monthly for new or worsening morphological abnormalities (eg, teardrop and nucleated red blood cells, immature egg white blood cells) or cytopenia(s).
- If the patient develops new or deterioration morphological abnormalities or cytopenia(s), discontinue treatment with RPLC195% and consider a bone marrow biopsy, including staining for fibrosis.
Worsened Thrombocytopenia After Cessation of Nplate
- Discontinuation of Nplate may result in thrombopenia of greater severity than was present prior to Nplate therapy. This worse thrombocytopenia crataegus laevigata increase the patient's risk of exposure of haemorrhage, particularly if Nplate is discontinued while the patient is on anticoagulants or antiplatelet agents.
- In clinical studies of patients with chronic ITP who had Nplate discontinued, four of 57 patients developed thrombocytopenia of greater severity than was deliver prior to Nplate therapy.
- This worsened thrombocytopenia resolved within 14 days.
- Following discontinuation of Nplate, obtain weekly CBCs, including platelet counts, for at least two weeks and consider alternative treatments for decline in quality thrombocytopenia, according to current treatment guidelines.
Thrombotic/thromboembolic Complications
- Thrombotic/thromboembolic complications may answer from unreasonable increases in platelet counts. Excessive doses of Nplate or medication errors that result in excessive Nplate doses whitethorn increase blood platelet counts to a level that produces thrombotic/thromboembolic complications. In controlled clinical studies, the incidence of thrombotic/thromboembolic complications was similar between Nplate and placebo.
- To minimize the risk for thrombotic/thromboembolic complications, do not use Nplate in an attempt to "normalize" blood platelet counts. Follow the dose adjustment guidelines to achieve and maintain a platelet count of ? 50 x 109/L.
Lack or Loss of Response to Nplate
- Hyporesponsiveness or failure to maintain a platelet response with Nplate should actuate a hunt for causative factors, including neutralizing antibodies to Nplate or bone marrow fibrosis.
- To detect antibody shaping, submit blood samples to Amgen (1-800-772-6436). Amgen will assay these samples for antibodies to Nplate and thrombopoietin (TPO).
- Discontinue Nplate if the platelet matter does non increase to a level sufficient to avoid clinically important hemorrhage after 4 weeks at the highest weekly dose of 10 mcg/kg.
Hematological Malignancies and Progression of Malignancy in Patients with a Pre-existing Hematological Malignancy or Myelodysplastic Syndrome (MDS)
- Nplate stimulation of the TPO receptor on the surface of hematopoietic cells may growth the jeopardy for haematological malignancies. In controlled clinical studies among patients with chronic ITP, the incidence of haematological malignancy was low and similar betwixt Nplate and placebo.
- In a separate single-arm clinical study of 44 patients with myelodysplastic syndromes (MDS), 11 patients were reported as having possible disease progression, among whom 4 patients had confirmation of acute myelogenous leukemia (AML) during follow-up.
- Nplate is not indicated for the treatment of thrombocytopenia due to MDS or whatever cause of thrombocytopenia early than chronic ITP.
Laboratory Monitoring
- Monitor CBCs, including platelet counts and peripheral blood smears, prior to initiation, end-to-end, and following discontinuation of Nplate therapy.
- Prior to the initiation of Nplate, examine the peripheral profligate differential to establish the baseline extent of redness and patrick White blood cell abnormalities.
- Obtain CBCs, including platelet counts and peripheral blood smears, weekly during the dosage adjustment phase of Nplate therapy and then monthly following ecesis of a stable Nplate dose. Obtain CBCs, including platelet counts, weekly for at least 2 weeks following discontinuation of Nplate.
Nplate Distribution Program
- Nplate is available only through and through a restricted distribution program called Nplate� NEXUS (Network of Experts Understanding and Supporting Nplate and Patients) Program. Under the Nplate� NEXUS Program, only prescribers and patients registered with the program are able to dictate, administer, and receive Nplate. This program provides educational materials and a mechanism for the proper function of Nplate. To enter in the Nplate� NEXUS Program, call off 1-877-NPLATE1 (1-877-675-2831).
General Safety
- In the placebo-controlled studies, headache was the most commonly reported adverse drug reaction, occurring in 35 percent of patients receiving Nplate and 32 percent of patients receiving placebo. Headaches were usually of mild or moderate rigour.
- Most coarse adverse reactions (? 5 per centum higher patient incidence in Nplate versus placebo) were Arthralgia (26 percent, 20 percent), Dizziness (17 percent, 0 pct), Insomnia (16 percent, 7 percent), Myalgia (14 percentage, 2 percentage), Pain in Extremity (13 percent, 5 percent) , Abdominal Pain (11 percent, 0 percent), Shoulder Pain (8 percent, 0 percent), Dyspepsia (7 pct, 0 per centum), and Paresthesia (6 pct, 0 per centum).
- As with all therapeutic proteins, patients may evolve antibodies to the sanative protein.
About Amgen
Amgen discovers, develops, manufactures and delivers modern human therapeutics. A bioengineering pioneer since 1980, Amgen was i of the first companies to actualize the new science's promise by bringing safe and effective medicines from research lab, to manufacturing plant, to patient. Amgen therapeutics have changed the practice of medicine, serving millions of people around the worldly concern in the fight against cancer, kidney disease, rheumatic arthritis and other serious illnesses. With a deep and unsubtle pipeline of potential new medicines, Amgen remains committed to forward-moving science to dramatically better people's lives. To learn more about our pioneering science and our full of life medicines, visit http://www.amgen.com.
Forward-Looking Statements
This news program release contains forward-looking statements that are based on management's current expectations and beliefs and are matter 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 modern statements, including estimates of revenues, in operation margins, capital expenditures, immediate payment, 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 need 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 nearly recent periodical 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 August 22, 2008 and expressly disclaims whatsoever duty to update information contained in this news release.
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