Genzyme in the UK

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New ‘Stem Cell Mobilisation’ Therapy Recommended by the All Wales Medicines Strategy Group

Date: April 8, 2010

Genzyme Welcomes Decision on Mozobil® (plerixafor) - a Therapy Which Offers Potential to Improve Management of Multiple Myeloma and Lymphoma Patients

Genzyme UK today welcomed the decision by the All Wales Medicines Strategy Group (AWMSG) recommending Mozobil® (plerixafor) for use within NHS Wales. Mozobil is a new ‘stem cell mobilisation’ therapy aimed at improving results for patients who are undergoing stem cell transplantation as part of their treatment for multiple myeloma and lymphoma. Mozobil quickly mobilises stem cells from the bone marrow into the blood stream for collection and offers hope for patients who might otherwise not be able to generate sufficient stem cells for a successful transplant. Mozobil has been shown to rapidly increase the number of stem cells available for collection in the blood stream.

This week’s announcement by the AWMSG follows the Scottish Medicines Consortium’s decision on use of Mozobil within NHS Scotland in January this year and positive recommendations by a number of individual health authorities in England.

Eric Low, Chief Executive of Myeloma UK, said: “This week’s decision is very good news for the relatively small numbers of myeloma patients in Wales who fail to mobilise stem cells following standard treatment. Mozobil increases the chances of this group having a successful collection and proceeding to transplant. We are delighted that both Wales and Scotland have now approved Mozobil as a clinically and cost-effective treatment for the NHS. We hope other decision-makers across the UK will ensure that myeloma patients who need Mozobil are also able to benefit from it.”

Dr Keith Wilson, Senior Clinical Lecturer in Haematology Cardiff University and Consultant Haematologist and Bone Marrow Transplant Director University Hospital of Wales, said: “Stem cell transplantation plays a major role in the effective treatment of both myeloma and lymphoma patients, allowing delivery of high dose chemotherapy. Historically, a proportion of patients would not have collected adequate numbers of stem cells using existing techniques to proceed to transplant. This week’s decision will, I hope, allow more patients to ultimately receive what can be lifesaving or life-prolonging treatment through stem cell transplantation.”

Mozobil was developed by Genzyme Corporation, one of the world’s leading biotechnology companies. In 2009, it received marketing authorisation from the European Medicines Agency (EMEA) for use in combination with granulocyte-colony stimulating factor to enhance mobilisation of haemopoietic stem cells to the peripheral blood for collection and subsequent transplantation in patients with lymphoma and multiple myeloma whose cells mobilise poorly. Mozobil has already been used in a number of UK hospitals on a named patient basis, for patients who had failed to mobilise sufficient stem cells for transplantation using current standard treatments.

Rory Cameron, Transplant and Oncology Business Unit Director for Genzyme UK, said: “We are driven by our commitment to developing and delivering highly effective therapies to improve the lives of patients suffering from life-threatening diseases. It is great news that patients in Wales will be able to benefit from Mozobil, an innovative new treatment that has the potential to make a real difference to the lives of those patients currently unable to harvest enough stem cells for effective transplant.”

About Mozobil and Autologous Stem Cell Transplantation

In treating myeloma and lymphoma, very high doses of chemotherapy potentially kill more cancer cells than standard doses and lead to a better overall response. However, normal bone marrow is badly damaged as part of the process bringing the risk of anaemia, infections and heavy bleeding. A transplant of healthy bone marrow cells offers a way round the problem. Mozobil is designed to be used with granulocyte-colony stimulating factor (G-CSF) to mobilise stem cells from a patient’s bone marrow into the blood stream where they can be collected, stored during the patient’s chemotherapy, and then returned to the patient once the chemotherapy has been completed. When infused into a patient’s bloodstream, stem cells travel to the marrow where they divide and develop into the three main types of cells found in blood (red cells, white cells and platelets).

Currently, however, approximately 19% of lymphoma and myeloma patients fail to collect the minimum number of cells required for autologous stem cell transplant at first attempt1.

Clinical studies of Mozobil in non-Hodgkin’s lymphoma and multiple myeloma have shown its use can significantly increase the proportion of patients who mobilise sufficient stem cells for transplant when compared to G-CSF alone. Fifty-seven percent of patients with non-Hodgkin’s lymphoma who received Mozobil and G-CSF collected the target number of stem cells compared with 19 percent of patients who received G-CSF alone, and demonstrated successful engraftment of their transplant2. In multiple myeloma, 70% of patients who received Mozobil and G-CSF collected the target number of stem cells compared to 34 percent of patients receiving G-CSF alone, and demonstrated successful engraftment of their transplant2. In the multiple myeloma trial, Mozobil also resulted in collection of stem cells rapidly with a median number of days to reach the target cell count of one day for the Mozobil group and four days for the control G-CSF alone group.


1. Pusic, I et al. Biology of Blood and Marrow Transplantation. 2008, 14: 1045-1056.

2. Mozobil (plerixafor injection) Summary of Product Characteristics. Naarden, The Netherlands: Genzyme Europe B.V.; 2009.

About Genzyme

One of the world's leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 12,000 employees in locations spanning the globe and 2009 revenues of $4.5 billion.

With many established products and services helping patients in approximately 100 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and immune disease, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as cardiovascular disease, neurodegenerative diseases, and other areas of unmet medical need.

Genzyme® is a registered trademark of Genzyme Corporation. All rights reserved.

Genzyme in the UK

Genzyme in the UK employs 600 people in research, development as well as commercial manufacturing and distribution roles. Genzyme UK comprises four different sites; Oxford, Kent, Haverhill and Cambridge.

Genzyme Therapeutics operates from the Oxford office and markets products for the treatment of patients with chronically debilitating diseases, like genetic disorders, such as lysosomal storage disorders (LSDs).

Genzyme Diagnostics UK is based on two sites at West Malling and Allington, near to Maidstone in Kent. Genzyme Diagnostics UK includes a Sales and Marketing group, Manufacturing Operations and a Research and Development Team.

Genzyme Haverhill Operations is located on the Cambridgeshire Suffolk border. The site has been a part of Genzyme since 1982, employs 350 people and has three distinct functions: cold chain supply centre, manufactures APIs and scale up and development of Genzyme’s new synthetic molecules.

Genzyme Europe Research is Genzyme’s first dedicated R&D facility in Europe. The science team is currently focused on Antibody Technology.


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Contact Info:

Genzyme Therapeutics Ltd
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Tel: +01865 405200
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Contact Info:

Genzyme Ltd, Haverhill Operations
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Tel: +01440 703522
Fax: +01440 716269