By a 10-5 Vote, ODAC Recommends REVLIMID as Oral Targeted Therapy for Low to
Intermediate-1-Risk MDS Patients With Deletion 5q Chromosomal Abnormality
WASHINGTON, Sept. 14 /PRNewswire-FirstCall/ -- Celgene Corporation
(Nasdaq: CELG) announced that the Oncologic Drugs Advisory Committee (ODAC) of
the U.S. Food and Drug Administration (FDA) recommended full approval of
REVLIMID (lenalidomide) for the treatment of patients with transfusion-
dependent anemia due to low- or intermediate-1-risk myelodysplastic syndromes
(MDS) associated with a deletion 5q cytogenetic abnormality with or without
additional cytogenetic abnormalities. The committee based its recommendation
on clinical data from an open label Phase II trial, evaluating REVLIMID in the
largest trial with MDS patients with deletion 5q chromosomal abnormality to
date. The data showed that:
-- Approximately two-thirds of patients achieved resolution of chronic
refractory anemia resulting in transfusion independence
-- Response was associated with meaningful cytogenetic and bone marrow
remission
-- Responder median hemoglobin increased more than 5.0 grams per
deciliter
-- After median follow-up of 58 weeks, the median duration of
transfusion-independence response had not yet been reached
-- The dosing in the study was based on tolerability and additional
studies are planned to refine dosing
-- The major side effects were cytopenias leading to dose reductions
"We are excited about ODAC's positive recommendation for approval of this
NDA. REVLIMID will offer the opportunity to improve the lives of deletion 5q
MDS patients with limited therapeutic options beyond blood transfusions," said
John W. Jackson, Chairman and Chief Executive Officer, Celgene Corporation.
About REVLIMID(R)
REVLIMID is a member of a new class of novel IMiDs(R), immunomodulatory
drugs. Celgene continues to evaluate treatments with REVLIMID for a broad
range of hematology and oncology conditions, including; multiple myeloma, the
malignant blood cell disorders known as myelodysplastic syndromes (MDS),
chronic lymphocytic leukemia as well as solid tumor cancers. REVLIMID affects
multiple intracellular biological pathways. The IMiD pipeline, including
REVLIMID(R), is covered by a comprehensive intellectual property estate of
U.S. and foreign issued and pending patent applications including composition-
of-matter and use patents.
About Myelodysplastic Syndromes
Myelodysplastic syndromes (MDS) 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. According to the American Cancer
Society, 10,000 to 20,000 new cases of MDS are diagnosed each year in the
United States, with mean survival rates ranging from approximately six months
to six years for the different classifications of MDS. MDS patients must 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.
About 5q Deletion Chromosomal Abnormality
Chromosomal (cytogenetic) abnormalities are detected in more than half of
patients with myelodysplastic syndrome (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 to 30% 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 ODAC
ODAC evaluated the REVLIMID NDA for the treatment of patients with
transfusion-dependent anemia due to low- or intermediate-1-risk
myelodysplastic syndromes (MDS) associated with a deletion 5q cytogenetic
abnormality with or without additional cytogenetic abnormalities. Both
Celgene and the FDA presented the results of the open label Phase II (MDS-003)
trial data.
These questions were voted upon by the committee:
Question #1: Randomized controlled trials allowed for direct comparisons
of treatment effects and safety between treatment arms. A single arm study
has been submitted using an 8-week run-in period to serve as a baseline for
each patient's transfusion requirements. A comparison is subsequently made to
a follow-up 8-week period on REVLIMID to compare transfusion requirements.
Does this study design allow adequate characterization of REVLIMID's treatment
effect in the population described in the proposed indication? Vote 11 Yes - 4
No
Question #2: In this single arm trial, 80% of patients enrolled in MDS-
003 has dose reductions and/or delays and 80% of patients experienced either
grade 3 or 4 adverse events. Data do not exist on the efficacy and safety of
lower REVLIMID doses. Approval of a drug is contingent upon being able to
write adequate product labeling, requiring a recommended dose and
characterization of a safety profile. Do the data provided in this single-arm
trial provide a basis for a recommendation dose and adequate description of a
safety profile? Vote 2 Yes -13 No
Question #3: Please characterize the magnitude of the benefit and risk of
REVLIMID(R) in the indication being sought. After this characterization, does
this risk/benefit analysis warrant approval? Vote 10 Yes -5 No
About Celgene
Celgene Corporation, headquartered in Summit, New Jersey, is an integrated
global pharmaceutical 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 http://www.celgene.com.
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 Corporation
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Related links:
http://www.celgene.com
CONTACT:
Robert J. Hugin, Senior VP and CFO,
+1-908-673-9102, or Brian P. Gill, Director PR/IR,
+1-908-673-9530, both of Celgene Corporation
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