Celgene Corporation announced that Revlimid has been granted orphan drug designation for myelodysplastic syndromes (MDS) by the European Commission (EC) following the favourable opinion of the European Agency for the Evaluation of Medicinal Products (EMEA). Revlimid is the lead development compound of a new class of novel immunomodulatory drugs, or IMiDs. Clinical trials, ongoing or soon to be initiated, are evaluating Revlimid in the treatment of a broad range of conditions, including; myelodysplastic syndromes, multiple myeloma, myelofibrosis, metastatic melanoma, renal cell carcinoma and ovarian cancer.
Orphan drug status is granted by the EC to promote development of drugs to treat rare diseases or conditions. Orphan drug designation may entitle Revlimid to ten years of market exclusivity for an indication in MDS; 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.
Myelodysplastic Syndromes are malignant disorders of blood cell production that eventually lead to acute leukemia and affect approximately 300,000 people worldwide making it one of the most common hematological abnormalities. Celgene's lead Immunomodulatory drug, Revlimid has received orphan drug and fast track designations from the US Food and Drug Administration (FDA) in February 2003 for the treatment of multiple 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 MDS supports our efforts to move Revlimid as quickly as possible through the clinical and regulatory development process worldwide," said Sol Barer, 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 MDS patients worldwide."
Revlimid is a member of a new class of novel immunomodulatory drugs, or IMiDs, 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 US 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.
Myelodysplastic syndromes (MDS) are a group of hematologic conditions that affect approximately 300,000 people worldwide. The five types of MDS are refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, refractory anemia with excess blasts in formation, and chronic myelomonocytic leukemia. Myelodysplastic syndromes occur when blood cells remain in an immature or "blast" stage within the bone marrow and never develop into mature cells capable of performing their necessary functions. Eventually, the bone marrow becomes filled with blast cells until there is no room for normal cells to develop. According to the American Cancer Society 14,000 new cases of MDS are diagnosed each year in the United States (approximately 87,000 worldwide), with survival rates ranging from six months to five years for the different types of MDS. MDS patients must now often rely on blood transfusions to manage symptoms of anemia and fatigue until they develop life-threatening iron overload and or toxicity, thus underscoring the critical need for new therapies targeting the cause of the condition rather than simply managing its symptoms.
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 fifteen 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.