Healthcare Industry News: U.S. Food and Drug Administration
News Release - November 10, 2017
Amgen Receives Positive CHMP Opinion To Expand Use Of Nplate(R) (romiplostim) In Pediatric Patients With Chronic Immune Thrombocytopenic PurpuraPositive Opinion Based on Five Studies Demonstrating Nplate Reduces Rates of Bleeding in Children With Rare Blood Disorder
THOUSAND OAKS, Calif., Nov. 10, 2017 -- (Healthcare Sales & Marketing Network) -- Amgen (NASDAQ: AMGN) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion to expand the current indication for NplateŽ (romiplostim) to include the treatment of chronic immune (idiopathic) thrombocytopenic purpura (ITP) for patients one year of age and older who are refractory to other treatments (e.g., corticosteroids, immunoglobulins).
The positive CHMP opinion was based on five studies evaluating the safety and efficacy of Nplate in children with ITP, including four completed studies (a Phase 3, a Phase 1/2 placebo-controlled study, and two long-term safety and efficacy studies) and one ongoing long-term safety and efficacy study.
"Children with ITP are at risk for serious bleeding events due to low platelet counts, and currently, limited therapeutic options are available to treat this rare disease," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "We are pleased by today's positive CHMP opinion and look forward to working with European regulatory authorities to deliver on our commitment to bring Nplate to pediatric patients with ITP who are suffering from thrombocytopenia."
ITP is a rare, serious autoimmune disease characterized by low platelet counts in the blood (a condition known as thrombocytopenia) and impaired platelet production.1,2 European studies report incidence rates in children between four and five cases per 100,000 children each year.3 The treatment goal for children with ITP is to promote a platelet count that maintains appropriate control of bleeding, improve symptoms and increase the number of platelets.1,4
The CHMP positive opinion will now be reviewed by the European Commission (EC), which has the authority to approve medicines for the European Union (EU). If approved, a centralized marketing authorization will be granted that will be valid in the 28 countries that are members of the EU. Norway, Iceland and Liechtenstein, as members of the European Economic Area (EEA), will take corresponding decisions on the basis of the decision of the EC.
About NplateŽ (romiplostim)
Nplate is a thrombopoietin (TPO) receptor agonist indicated for the treatment of low blood platelet counts in adults with chronic ITP, who had an insufficient response to other medicines or surgery. Nplate mimics the body's natural thrombopoietin and is designed to increase platelet counts in patients with chronic ITP.5
Nplate is the first U.S. Food and Drug Administration (FDA)-approved treatment specifically for adult chronic ITP.
In the U.S., Nplate is indicated for the treatment of thrombocytopenia in patients with chronic ITP 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 chronic 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.
In the EU, Nplate is indicated for the treatment of adult chronic ITP patients who are refractory to other treatments (e.g., corticosteroids, immunoglobulins).
Nplate was named as a recipient of the U.S. Prix Galien 2009 "Best Biotechnology Product" award and also received the 2009 Scrip Awards for "Best New Drug." Nplate has also been honored with numerous awards throughout the EU, including a 2010 Prix Galien in France in the category of "Drugs for Rare Diseases," and the 2011 Prix Galien in Germany in the category of "Specialist Care." In September 2010, Nplate was awarded the 2010 International Prix Galien Award, an award granted every two years which recognizes the "Best of the Best" selected from previous national Prix Galien award recipients.
Nplate is also approved in Canada, Australia, Russia, Mexico, Switzerland, Lichtenstein, Japan, Argentina, Israel, South Korea, Hong Kong, Chile, Serbia, Kazakhstan, Malaysia, Singapore, Colombia, Kuwait, Taiwan, South Africa, Brazil, Guatemala, Morocco, Ecuador, Macau, Egypt, Lebanon, Peru and Venezuela. Nplate has received orphan designation for chronic ITP in the U.S. (2003), the EU (2005), Switzerland (2005), Japan (2006), Mexico and South Korea (2010).
For more information about Nplate, please visit www.Nplate.com.
Important U.S. NplateŽ Safety Information
Risk of Progression of Myelodysplastic Syndromes to Acute Myelogenous Leukemia
- In NplateŽ clinical trials of patients with myelodysplastic syndromes (MDS) and severe thrombocytopenia, progression from MDS to acute myelogenous leukemia (AML) has been observed.
- NplateŽ is not indicated for the treatment of thrombocytopenia due to MDS or any cause of thrombocytopenia other than chronic ITP.
- Thrombotic/thromboembolic complications may result from increases in platelet counts with NplateŽ use. Portal vein thrombosis has been reported in patients with chronic liver disease receiving NplateŽ.
- To minimize the risk for thrombotic/thromboembolic complications, do not use NplateŽ in an attempt to normalize platelet counts. Follow the dose adjustment guidelines to achieve and maintain a platelet count of ≥ 50 x 109/L.
- Hyporesponsiveness or failure to maintain a platelet response with NplateŽ should prompt a search for causative factors, including neutralizing antibodies to NplateŽ.
- To detect antibody formation, submit blood samples to Amgen (1-800-772-6436). Amgen will assay these samples for antibodies to NplateŽ and thrombopoietin (TPO).
- Discontinue NplateŽ if the platelet count does not increase to a level sufficient to avoid clinically important bleeding after 4 weeks at the highest weekly dose of 10 mcg/kg.
- Obtain CBCs, including platelet counts, weekly during the dose adjustment phase of NplateŽ therapy and then monthly following establishment of a stable NplateŽ dose.
- Obtain CBCs, including platelet counts, weekly for at least two weeks following discontinuation of NplateŽ.
- In the placebo-controlled trials, headache was the most commonly reported adverse drug reaction, occurring in 35% of patients receiving NplateŽ and 32% of patients receiving placebo. Headaches were usually of mild or moderate severity.
- Most common adverse reactions (≥ 5% higher patient incidence in NplateŽ versus placebo) were Arthralgia (26%, 20%), Dizziness (17%, 0%), Insomnia (16%, 7%), Myalgia (14%, 2%), Pain in Extremity (13%, 5%), Abdominal Pain (11%, 0%), Shoulder Pain (8%, 0%), Dyspepsia (7%, 0%), and Paresthesia (6%, 0%).
- NplateŽ administration may increase the risk for development or progression of reticulin fiber formation within the bone marrow. This formation may improve upon discontinuation of NplateŽ. In a clinical trial, one patient with ITP and hemolytic anemia developed marrow fibrosis with collagen during NplateŽ therapy.
- Women who become pregnant during NplateŽ treatment are encouraged to enroll in Amgen's Pregnancy Surveillance Program. Patients or their physicians should call 1‑800‑77‑AMGEN (1‑800‑772‑6436) to enroll.
Important EU NplateŽ Safety Information
The EU Summary of Product Characteristics for NplateŽ lists the following Special Warnings and Precautions: reoccurrence of thrombocytopenia and bleeding after cessation of treatment, increased bone marrow reticulin, thrombotic/thromboembolic complications, progression of existing myelodysplastic syndromes (MDS), medication errors, loss of response to Nplate, and effects on red and white blood cells.
The most common adverse reactions observed include hypersensitivity reactions (including cases of rash, urticaria and angioedema) and headache. As with all therapeutic proteins, there is a potential for immunogenicity.
Please refer to the Summary of Product Characteristics for full European prescribing information.
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.
For more information, visit www.amgen.com and follow us on www.twitter.com/amgen.
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The scientific information discussed in this news release relating to new indications for our products is preliminary and investigative and is not part of the labeling approved by the U.S. Food and Drug Administration for the products. The products are not approved for the investigational use(s) discussed in this news release, and no conclusions can or should be drawn regarding the safety or effectiveness of the products for these uses.
- Patient. Immune Thrombocytopenia. http://patient.info/health/immune-thrombocytopenia-leaflet. Accessed Aug. 29, 2017.
- Cines DB et al. Immune thrombocytopenic purpura. N Engl J Med. 2002;346:995-1008.
- Segal JB, Powe NR. Prevalence of immune thrombocytopenia: analyses of administrative data. J Thromb Haemost. 2006;4(11):2377-83.
- US National Institutes of Health. Immune Thrombocytopenia. http://www.nhlbi.nih.gov/book/export/html/4917. Accessed Aug. 29, 2017.
- NplateŽ (romiplostim) prescribing information, Amgen.
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