Mozobil - plerixafor

What is Mozobil?

Mozobil is a solution for injection containing the active substance plerixafor.

What is Mozobil used for?

Mozobil is used to help collect hematopoietic stem cells (cells in the bone marrow that can mutate 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 used only in patients in whom stem cell collection is difficult.

Since the number of patients requiring mobilization and collection of hematopoietic stem cells is low, this disease is considered "rare", so Mozobil was designated an "orphan medicine" (a medicine used in rare diseases) on 20 October 2004.

How is Mozobil used?

Treatment with Mozobil should be initiated and supervised by a doctor experienced in oncology or hematology. After administering Mozobil to the patient, it is necessary to extract the patient's stem cells from the blood and store them before transplantation. Therapy must therefore be performed in collaboration with a specialized center with experience in this field and in which stem cell monitoring can be carried out 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 the addition of Mozobil. Mozobil

it is administered by subcutaneous injection, six to eleven hours before each patient's blood sampling session and stem cell extraction. It can be used up to seven consecutive days.

How does Mozobil work?

Mozobil is used to help move ("mobilize") stem cells from the bone marrow so that they can be released into the blood. Plerixafor, the active ingredient present in Mozobil, acts by inhibiting the activity of a protein called "CXCR4 chemokine receptor". This protein helps retain stem cells in the bone marrow. By blocking its activity, Mozobil allows stem cells to be released into the blood, so they can be harvested.

What studies have been carried out on Mozobil?

The effects of Mozobil were first tested in experimental models before being studied in humans.

Mozobil was 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 patients were also given G-CSF. The main efficacy parameter was the number of patients in which it was possible to collect a specific number of stem cells from the blood in 2-4 days of collection. Furthermore, in the studies the number of patients with a specific number of stem cells collected was observed and in which the stem cells were successfully grafted (starting to grow normally and produce blood cells).

What benefit has Mozobil shown during the studies?

Mozobil was more effective than placebo in mobilizing stem cells from bone marrow in the blood. Among lymphoma patients, 60% of those given Mozobil achieved the numeric target of stem cells within 4 days of collection (89 out of 150), compared to 20% of patients who received placebo (29 on 148). Among patients with multiple myeloma, 72% of those who received Mozobil achieved the numeric target of stem cells (106 out of 148), compared to 34% of patients who received placebo (53 out of 154). In both studies there were a greater number of patients who received Mozobil who achieved the numeric target of stem cells and where the stem cells were successfully grafted.

What is the risk associated with Mozobil?

The most common side effects of Mozobil (seen in more than 1 patient in 10) are diarrhea, nausea and injection site reactions. 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 improve hematopoietic stem cell mobilization to peripheral blood for collection and subsequent autologous transplantation in patients with lymphoma and multiple myeloma with poor cell mobilization. The committee recommended the granting of the marketing authorization for Mozobil.