Healthcare Industry News:  myelodysplastic syndromes 

Biopharmaceuticals Oncology Regulatory

 News Release - February 19, 2008

Lenalidomide (REVLIMID(R)) Receives Orphan Drug Status in Japan for Multiple Indications

Ministry of Health, Labour and Welfare grants designation in both deletion 5q MDS and previously treated multiple myeloma

BOUDRY, Switzerland--(HSMN NewsFeed)--Celgene International Sŕrl (NASDAQ: CELG ) announced that lenalidomide (CC-5013) has been granted orphan drug status by Japan’s Ministry of Health, Labour and Welfare (MHLW) for two different indications: the treatment of both anemia due to low- or intermediate-1-risk myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenic abnormality with or without other cytogenic abnormalities; and in combination with dexamethasone for multiple myeloma patients who have received at least one prior therapy.

Orphan drug status is granted by the MHLW to promote development of drugs to treat rare diseases or conditions. Such designation confers multiple incentives for development, including access to a fast-track Marketing Authorization Approval procedure; financial incentives for development expenses; protocol development assistance; and a ten-year registration validity period once the product is approved.

“The decision by the MHLW to grant lenalidomide orphan drug status for both relapsed/refractory multiple myeloma and deletion 5q MDS represents a major step in our efforts to deliver REVLIMID as a therapeutic option for patients living with these conditions worldwide as quickly as possible,” said Graham Burton M.D., SVP, Global Regulatory Affairs and Pharmacovigilance for Celgene Corporation. “We will continue to work closely with the Japanese regulatory authorities as we move lenalidomide through the clinical development process.”

Lenalidomide (REVLIMID) has obtained orphan drug designation for MDS in the European Union (EU), United States (US), and Australia, orphan drug designation for multiple myeloma (MM) in the EU, US, Australia and Switzerland, and orphan drug designation for chronic lymphocytic leukemia (CLL) in the EU and US. REVLIMID is currently approved for use in the US and Canada for the treatment of patients with transfusion-dependent anemia due to low- or intermediate-1-risk MDS associated with a deletion 5q cytogenetic abnormality with or without additional cytogenetic abnormalities. REVLIMID is also approved for use in the EU, US and Switzerland for the treatment of multiple myeloma in combination with dexamethasone in patients who have received at least one prior therapy, and Australia for patients with multiple myeloma whose disease has progressed after one therapy.

About REVLIMID®

REVLIMID is an IMiDs® compound, a member of a proprietary group of novel immunomodulatory agents. REVLIMID and other IMiDs compounds continue to be evaluated in over 100 clinical trials in a broad range of hematological and oncological conditions. The IMiDs pipeline is covered by a comprehensive intellectual property estate of issued and pending patent applications in the US, EU and other regions, including composition-of-matter and use patents.

About Multiple Myeloma

Multiple myeloma (also known as myeloma or plasma cell myeloma) is the second most commonly diagnosed blood cancer. According to the International Myeloma Foundation, there are an estimated 750,000 people with MM worldwide. Multiple 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 remains unknown.

About myelodysplastic syndromes

myelodysplastic syndromes are a group of hematologic malignancies that affect approximately 300,000 people worldwide. 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 may be filled with blast cells suppressing normal cell development. MDS patients must often rely on blood transfusions to manage symptoms of anemia and fatigue and may develop life-threatening iron overload and/or toxicity from frequent transfusions, thus underscoring the critical need for new therapies targeting the cause of the condition rather than simply managing its symptoms.

About Deletion 5q Chromosomal Abnormality

Chromosomal (cytogenetic) abnormalities are detected in more than half of patients with MDS, and involve a deletion in all or part of one or more specific chromosomes. The most common cytogenetic abnormalities in MDS are deletions in the long arm of chromosomes 5, 7, and 20. Another common abnormality is an extra copy of chromosome 8. A deletion involving the 5q chromosome may be involved in 20 percent to 30 percent of all MDS patients. The World Health Organization has also recently identified a unique subset of MDS patients with a "5q- Syndrome" where the only chromosomal abnormality is a specific portion of the 5q chromosome.

About Celgene International Sárl

Celgene International Sárl, located in Neuchâtel, Switzerland, is a wholly owned subsidiary and international headquarters of Celgene Corporation. Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through gene and protein regulation. For more information, please visit the Company's website at www.celgene.com.

REVLIMID® is a registered trademark of Celgene Corporation.

This release contains forward-looking statements which are subject to known and unknown risks, delays, uncertainties and other factors not under the Company's control, which may cause actual results, performance or achievements of the Company to be materially different from the results, performance or other expectations expressed or implied by these forward-looking statements. These factors include results of current or pending research and development activities, actions by the FDA and other regulatory authorities, and other factors described in the Company's filings with the Securities and Exchange Commission such as our 10K, 10Q and 8K reports.


Source: Celgene International Sarl

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