Active Substance: romiplostim
Common Name: romiplostim
ATC Code: B02BX04
Marketing Authorisation Holder: Amgen Europe B.V.
Active Substance: romiplostim
Authorisation Date: 2009-02-04
Therapeutic Area: Purpura, Thrombocytopenic, Idiopathic
Pharmacotherapeutic Group: Antihaemorrhagics
Nplate is indicated for adult chronic-immune (idiopathic)-thrombocytopenic-purpura (ITP) splenectomised patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins).
Nplate may be considered as second-line treatment for adult non-splenectomised patients where surgery is contra-indicated.
What is Nplate?
Nplate is a powder that is made up into a solution for injection. Nplate is available with or without a solvent. It contains the active substance romiplostim.
What is Nplate used for?
Nplate is used in adults with long-term immune thrombocytopenic purpura (ITP), a disease in which the patientâ€™s immune system destroys the platelets (components in the blood that help it to clot). Patients with ITP have low platelet counts and are at risk of bleeding.
Nplate is used in patients who have already been treated with medicines such as corticosteroids or immunoglobulins and who have had their spleen removed, if these treatments have not worked. It can also be considered for use in patients who have been treated for ITP who have a spleen and cannot have surgery. The spleen is an organ that is involved in the destruction of platelets.
Because the number of patients with ITP is low, the disease is considered â€˜rareâ€™, and Nplate was designated an â€˜orphan medicineâ€™ (a medicine used in rare diseases) on 27 May 2005.
The medicine can only be obtained with a prescription.
How is Nplate used?
Treatment with Nplate should be supervised by a doctor who has experience in treating blood diseases.
Nplate is given once a week as an injection under the skin. The starting dose depends on the patientâ€™s weight, and is then adjusted every week to maintain platelet counts at target levels. Treatment can be interrupted if platelet counts become too high. Treatment with Nplate should be stopped after four weeks of treatment with the maximum dose of Nplate if the platelet count does not reach levels that are high enough to reduce the risk of bleeding. Some patients whose platelet levels have stabilised may be able to administer the medicine themselves after they have been properly trained.
How does Nplate work?
The active substance in Nplate, romiplostim, is a medicine that stimulates the production of platelets. In the body, a hormone called â€˜thrombopoietinâ€™ stimulates the production of platelets in the bone marrow. Romiplostim is a protein that has been â€˜engineeredâ€™ (specifically designed) so that it can attach to and stimulate the same receptors as thrombopoietin. By mimicking the action of thrombopoietin, romiplostim stimulates the production of platelets, increasing blood platelet counts.
Romiplostim is produced by a method known as â€˜recombinant DNA technologyâ€™: it is made by bacteria into which a gene (DNA) has been introduced which makes them able to produce romiplostim.
How has Nplate been studied?
Nplate has been compared with placebo (a dummy treatment) in two main studies involving adults with long-term ITP. The first study involved 63 patients whose spleens had been removed but whose disease was still not controlled. The second study involved 62 patients who still had their spleens and who had been treated for ITP in the past.
In both studies, the main measure of effectiveness was the number of patients who had a lasting response to treatment. This was classified as the patientâ€™s platelet counts being above 50 million per millilitre for at least six of the last eight weeks of the 24-week treatment period, without the need for any other medicines for ITP. Platelet counts below 30 million per millilitre are considered to put ITP patients at risk of bleeding, while normal counts are between 150 and 400 million per millilitre.
What benefit has Nplate shown during the studies?
Nplate was more effective than placebo at increasing blood platelet counts. In the study of patients whose spleen had been removed, 38% of the patients had a lasting response to treatment with Nplate (16 out of 42), compared with none of the 21 patients receiving placebo. In the study of patients with a spleen, 61% of the patients had a lasting response to treatment with Nplate (25 out of 41), compared with 5% of the patients receiving placebo (1 out of 21).
What is the risk associated with Nplate?
The most common side effects with Nplate (seen in more than 1 patient in 10) are headache and hypersensitivity (allergic reactions). For the full list of all side effects reported with Nplate, see the package leaflet.
Nplate must not be used in people who are hypersensitive (allergic) to romiplostim, any of the other ingredients, or proteins produced by Escherichia coli (a bacterium).
Why has Nplate been approved?
The CHMP noted that the effectiveness of Nplate had been shown in patients whose spleens had been removed, as well as in patients who still had their spleens. However, because removal of the spleen is a potential cure for ITP, the Committee concluded that Nplate should only be used in patients with a spleen if they are not able to undergo surgery. The CHMP decided that Nplateâ€™s benefits are greater than its risks and recommended that it be given marketing authorisation.
What information is still awaited for Nplate?
The company that makes Nplate will provide educational packs for doctors in each Member State. The packs will include information on the safety and effectiveness of Nplate. They will also remind doctors how to use the medicine and to discuss the benefits and risks of Nplate with patients. Doctors will also receive a â€˜dosing calculatorâ€™ to help them calculate the volumes of Nplate that need to be injected. These volumes can sometimes be very small.
Other information about Nplate
The European Commission granted a marketing authorisation valid throughout the European Union for Nplate on 4 February 2009.
For more information about treatment with Nplate, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.
Source: European Medicines Agency
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