Active Substance: plerixafor
Common Name: plerixafor
ATC Code: L03AX16
Marketing Authorisation Holder: Genzyme Europe B.V.
Active Substance: plerixafor
Authorisation Date: 2009-07-31
Therapeutic Area: Multiple Myeloma Hematopoietic Stem Cell Transplantation Lymphoma
Pharmacotherapeutic Group: Immunostimulants
Mozobil is indicated in combination with granulocyte-colony-stimulating factor to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with lymphoma and multiple myeloma whose cells mobilise poorly (see section 4.2).
What is Mozobil?
Mozobil is a solution for injection that contains the active substance plerixafor.
What is Mozobil used for?
Mozobil is used to help to collect haematopoietic stem cells (cells in the bone marrow that can develop into different types of blood cells) for transplantation. It is used in patients with lymphoma or multiple myeloma (types of blood-cell cancer) for autologous transplantation (when the patient’s own cells are used in the transplant). It is only used in patients in whom collection of stem cells is difficult.
Because the number of patients that need mobilisation and collection of haematopoietic stem cells for transplantation is low, the condition is considered ‘rare’, and Mozobil was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 20 October 2004.
How is Mozobil used?
Treatment with Mozobil should only be started and supervised by a doctor who has experience in treating cancer or blood disorders. After the patient has been given Mozobil, the patient’s stem cells need to be extracted from the blood and stored before transplantation. Because of this, treatment should be carried out in collaboration with a specialised centre that has experience with this type of procedure and can monitor the stem cells correctly.
Mozobil is used together with a hormone called granulocyte-colony-stimulating factor (G-CSF). G-CSF is used on its own for four days before Mozobil is added. Mozobil is given as an injection under the skin, six to 11 hours before each session when the patient’s blood is taken and the stem cells are extracted. It can be used for up to seven consecutive days.
How does Mozobil work?
Mozobil is used to help dislodge (‘mobilise’) the stem cells from the bone marrow so they can be released into the blood. The active substance in Mozobil, plerixafor, works by blocking the activity of a protein called the ‘CXCR4 chemokine receptor’. This protein normally helps to keep stem cells within the bone marrow. By blocking its activity, Mozobil allows the stem cells to be released into the blood, so that they can be collected.
How has Mozobil been studied?
The effects of Mozobil were first tested in experimental models before being studied in humans.
Mozobil has been compared with placebo (a dummy treatment) in two main studies involving 298 adults with a type of lymphoma called non-Hodgkin’s lymphoma and 302 adults with multiple myeloma. All of the patients were also receiving G-CSF. The main measure of effectiveness was the number of patients in whom a target number of stem cells could be collected from the blood within two or four collection days. The studies also looked at the number of patients with a target number of stems cells collected and in whom the stems cells were successfully engrafted (started growing and producing normal blood cells).
What benefit has Mozobil shown during the studies?
Mozobil was more effective than placebo at mobilising stems cells from the bone marrow into the blood. Among the patients with lymphoma, 60% of those receiving Mozobil achieved the target number of stems cells within four collection days (89 out of 150), compared with 20% of the patients receiving placebo (29 out of 148). Among the patients with multiple myeloma, 72% of those receiving Mozobil achieved the target number of stem cells (106 out of 148), compared with 34% of the patients receiving placebo (53 out of 154). In both studies, there were more patients who received Mozobil that achieved the target number of stem cells and in whom the stems cells were successfully engrafted.
What is the risk associated with Mozobil?
The most common side effects with Mozobil (seen in more than 1 patient in 10) are diarrhoea, nausea (feeling sick) and reactions at the site of injection. For the full list of all side effects reported with Mozobil, see the package leaflet.
Mozobil should not be used in people who may be hypersensitive (allergic) to plerixafor or any of the other ingredients.
Why has Mozobil been approved?
The Committee for Medicinal Products for Human Use (CHMP) decided that Mozobil’s benefits are greater than its risks for use in combination with G-CSF to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with lymphoma and multiple myeloma whose cells mobilise poorly. The Committee recommended that Mozobil be given marketing authorisation.
Other information about Mozobil
The European Commission granted a marketing authorisation valid throughout the European Union for Mozobil to Genzyme Europe B.V. on 31 July 2009.
Source: European Medicines Agency
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