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Revlimid Receives Orphan Drug Designation From the European Commission for Multiple Myeloma
Orphan drug status is granted by the EC to promote development of drugs to treat rare diseases or conditions. Orphan drug designation will entitle REVLIMID to ten years of market exclusivity for an indication in multiple myeloma; protocol assistance by EMEA to optimize drug development in preparing a dossier that will meet regulatory requirements; facilitate access to the Centralized Procedure for the application for marketing approval; reduce fees associated with applying for marketing approval and protocol assistance; and secure a European Union research funding grant.
Multiple myeloma is the second most common blood cancer worldwide affecting approximately 150,000 people. About 74,000 new cases of multiple myeloma are diagnosed each year and an estimated 57,000 deaths were reported worldwide in 2002. Celgene's lead Immunomodulatory drug, REVLIMID has received orphan drug and fast track designations from the U.S. Food and Drug Administration (FDA) in February 2003 for the treatment of myeloma and in April 2003 for the treatment of myelodysplastic syndromes (MDS), a malignant disorder of blood cell production that eventually lead to acute leukemia and affect approximately 300,000 people worldwide.
"The decision by the European Commission to grant REVLIMID orphan drug status for multiple myeloma supports our efforts to move REVLIMID as quickly as possible through the clinical and regulatory development process worldwide," said Sol J. Barer, Ph.D., President and Chief Operating Officer of Celgene Corporation. "We continue to be encouraged by the growing body of published and presented data on REVLIMID by key opinion leaders at major medical meetings, and based on these findings, we are committed to accelerating wherever possible our efforts to help address the unmet medical needs of myeloma patients worldwide."
REVLIMID is a member of a new class of novel immunomodulatory drugs, or IMiDs (TM), which may demonstrate, in clinical studies, anticancer response. Ongoing clinical trials are evaluating REVLIMID in the treatment of a broad range of conditions, including; multiple myeloma, the malignant blood cell disorders known as myelodysplastic syndromes (MDS), metastatic melanoma, as well as solid tumor cancers. REVLIMID is believed to affect multiple biological pathways within the cell, which ultimately may be responsible for the clinical response observed in multiple cancer studies. The IMiD pipeline, including REVLIMID, is covered by a comprehensive intellectual property estate of U.S. and foreign issued patents and pending patent applications including composition-of-matter and use patents.
REVLIMID (CC-5013) is not approved by the FDA or any other regulatory agencies as a treatment in any indication and is currently being evaluated in clinical trials for efficacy and safety for future regulatory applications.
About Multiple Myeloma
Multiple myeloma (also known as myeloma or plasma cell myeloma) is a cancer of the blood in which malignant plasma cells are overproduced in the bone marrow. Plasma cells are white blood cells that help produce antibodies called immunoglobulins that fight infection and disease. However, most patients with multiple myeloma have cells that produce a form of immunoglobulin called paraprotein (or M protein) that does not benefit the body. In addition, the malignant plasma cells replace normal plasma cells and other white blood cells important to the immune system. Multiple myeloma cells can also attach to other tissues of the body, such as bone, and produce tumors. The cause of the disease is unknown. Multiple myeloma is the second most common cancer of the blood, representing approximately one percent of all cancers and two percent of all cancer deaths with a worldwide prevalence of approximately 150,000 cases. In the year 2002, there were an estimated 74,000 new cases of multiple myeloma worldwide. The estimated number of deaths from multiple myeloma in 2002 was 57,370 worldwide.
The European Agency for the Evaluation of Medicinal Products (EMEA) is the European regulatory body responsible for the authorization and supervision of medicinal products for human and veterinary use in member European countries, approximately twenty-seven to date. The agency has four key objectives; (1) To protect public health by mobilizing the best scientific resources existing within the European Union, (2) To promote health care through the effective regulation of new pharmaceuticals and better information for users and health professionals, (3) To facilitate quicker access and the free circulation of pharmaceuticals within the European single market, (4) To support the European pharmaceutical research and development industry by developing efficient, effective and responsive operating procedures.
Source: Celgene Corporation