Celgene International Sarl (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(R)
REVLIMID is an IMiDs(R) 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 Sarl
Celgene International Sarl, located in Neuchatel, 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(R) 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.
