"These new data are the first of their kind to prospectively examine treatment-free remission as an outcome for patients with ITP. Thirty-two percent of patients who received Nplate soon after an insufficient response to the first course of steroids maintained platelet response for at least six months without Nplate or any other ITP therapy," said
The sBLA was based on an open-label, single-arm Phase 2 trial of adults with ITP diagnosed ≤ 6 months prior who had an insufficient response to first-line treatment, including corticosteroids (N=75). The median time from ITP diagnosis to study enrollment was 2.2 months. On the primary endpoint, the median number of months with platelet response (≥ 50 x 109/L) was 11 months during the 12-month treatment period (95% CI: 10, 11), with a median time to first platelet response of 2.1 weeks (95% CI: 1.1, 3.0). Additionally, 93% (70) of patients achieved one or more platelet responses during the 12-month treatment period. On the secondary endpoint, 32% (24) of patients achieved remission for at least six months, defined by maintaining a platelet count ≥ 50 x 109/L in the absence of Nplate and any medication for ITP (concomitant or rescue).
"Among adults with immune thrombocytopenia, there is a need for treatment options that can get patients to sustained remission," said
The safety profile of Nplate was similar across patients, regardless of ITP duration. The following adverse reactions (at least 5% incidence and at least 5% more frequent with Nplate compared with placebo or standard of care) occurred in Nplate patients with ITP duration up to 12 months: bronchitis, sinusitis, vomiting, arthralgia, myalgia, headache, dizziness, diarrhea, upper respiratory tract infection, cough, nausea and oropharyngeal pain. The adverse reaction of thrombocytosis occurred with an incidence of 2% in adults with ITP duration up to 12 months.
About the Phase 2 Study
The Phase 2 study was a single-arm, open-label study designed to assess the safety and efficacy of Nplate in adult patients who had an insufficient response (platelet count ≤ 30 x 109/L) to first line therapy (N=75). The median time from ITP diagnosis to study enrollment was 2.2 months. Prior ITP treatments included corticosteroids, immunoglobulins and anti-D immunoglobulins. Rescue therapies were permitted. Patients received single weekly SC injections of Nplate over a 12-month treatment period, with individual dose adjustments to maintain platelet counts (50 x 109/L to 200 x 109/L).
About Immune Thrombocytopenia (ITP)
ITP is a rare, serious autoimmune disease characterized by low platelet counts in the blood (a condition known as thrombocytopenia) and impaired platelet production.1 In the U.S., the estimated incidence of ITP is 6.1 per 100,000 adults annually.2 Nearly 20,000 people are newly diagnosed with ITP each year in the U.S.2
About Nplate® (romiplostim)
Nplate is a thrombopoietin (TPO) receptor agonist that mimics the body's natural TPO and is designed to increase platelet counts in patients with ITP.3
In the U.S:
Nplate is also approved in 69 countries, including Canada and Australia.
For more information about Nplate, please visit www.Nplate.com.
IMPORTANT SAFETY INFORMATION
Risk of Progression of Myelodysplastic Syndromes to Acute Myelogenous Leukemia
Loss of Response to Nplate®
Nplate® administration may increase the risk for development or progression of reticulin fiber formation within the bone marrow. This formation may improve upon discontinuation of Nplate®. In a clinical trial, one patient with ITP and hemolytic anemia developed marrow fibrosis with collagen during Nplate® therapy.
Nplate® is a thrombopoietin receptor agonist indicated for the treatment of thrombocytopenia in adult patients with immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. Nplate® is indicated for the treatment of thrombocytopenia in pediatric patients 1 year of age and older with ITP for at least 6 months who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
Nplate® is not indicated for the treatment of thrombocytopenia due to myelodysplastic syndrome (MDS) or any cause of thrombocytopenia other than ITP. Nplate® should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding. Nplate® should not be used in an attempt to normalize platelet counts.
Please see full Prescribing Information and Medication Guide.
About Amgen Oncology
For the last four decades, we have been dedicated to discovering the firsts that matter in oncology and to finding ways to reduce the burden of cancer. Building on our heritage,
For more information, follow us on www.twitter.com/amgenoncology.
Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.
Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people's lives. A biotechnology pioneer since 1980, Amgen has grown to be one of the world's leading independent biotechnology companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.
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