FDA Fast Track Designation Granted to Luspatercept for the Treatment of Patients with Beta-Thalassemia
May 18 2015 - 7:30AM
Business Wire
Celgene Corporation (NASDAQ:CELG) and Acceleron Pharma Inc.
(NASDAQ:XLRN) today announced that the United States Food and Drug
Administration (FDA) has granted Fast Track designations to
luspatercept for two separate indications; the use of luspatercept
for the treatment of patients with transfusion dependent
beta-thalassemia and the use of luspatercept for the treatment of
patients with non-transfusion dependent beta-thalassemia. Celgene
and Acceleron are jointly developing luspatercept.
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The Fast Track program of the FDA is designed to facilitate the
development and expedite the review of new drugs that are intended
to treat serious or life-threatening conditions and that
demonstrate the potential to address unmet medical needs. The
designation also provides the opportunity for more frequent
interaction with FDA over the course of the development program,
and allows a sponsor to submit sections of the BLA on a rolling
basis as they are finalized.
“The FDA’s Fast Track designation for the luspatercept
development program recognizes the serious unmet medical needs of
patients with beta-thalassemia and the potential for luspatercept
in this area,” said Jacqualyn A. Fouse, President,
Hematology/Oncology for Celgene. “Celgene and Acceleron are working
diligently to initiate a phase 3 clinical program in 2015 to treat
patients with beta-thalassemia and we look forward to continuing to
work closely with health authorities and other important
stakeholders to advance this program.”
About Luspatercept
Luspatercept is a modified activin receptor type IIB fusion
protein that acts as a ligand trap for members of the Transforming
Growth Factor-Beta (TGF-beta) superfamily involved in the late
stages of erythropoiesis (red blood cell production). Luspatercept
regulates late-stage erythrocyte (red blood cell) precursor cell
differentiation and maturation. This mechanism of action is
distinct from that of erythropoietin (EPO), which stimulates the
proliferation of early-stage erythrocyte precursor cells. Acceleron
and Celgene are jointly developing luspatercept as part of a global
collaboration. Luspatercept is currently in phase 2 clinical trials
in patients with beta-thalassemia and in patients with
myelodysplastic syndromes. For more information, please visit
www.clinicaltrials.gov.
About Beta-thalassemia
Beta-thalassemia is an inherited disease involving mutations in
the beta globin gene leading to deficient hemoglobin levels and
defective red blood cell (RBC) production in the bone marrow known
as ineffective erythropoiesis. Beyond the severe anemia, many
patients also suffer from multiple organ dysfunction, largely due
to excess iron deposits, known as "iron overload," resulting from
the ineffective erythropoiesis as well as the repeated RBC
transfusions to address the anemia. Iron overload can lead to heart
failure, liver fibrosis, and diabetes, among other consequences.
Current clinical management for beta-thalassemia includes RBC
transfusions and iron chelation therapy, which is associated with
toxicities. There are no drugs approved to treat beta-thalassemia
leaving healthcare providers with few options for patients.
About Acceleron
Acceleron is a clinical stage biopharmaceutical company focused
on the discovery, development and commercialization of novel
therapeutic candidates that regulate cellular growth and repair.
The company is a leader in understanding the biology of the
TGF-beta protein superfamily, a large and diverse group of
molecules that are key regulators in the growth and repair of
tissues throughout the human body, and in targeting these pathways
to develop important new medicines. Acceleron has built a highly
productive R&D platform that has generated innovative clinical
and preclinical protein therapeutic candidates with novel
mechanisms of action. These therapeutic candidates have the
potential to significantly improve clinical outcomes for patients
with cancer and rare diseases.
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 www.celgene.com. Follow Celgene on Social Media:
@Celgene, Pinterest, LinkedIn and YouTube.
Forward-Looking Statements
Acceleron:
Cautionary Note on Forward-Looking Statements
This press release includes forward-looking statements about
Acceleron's strategy, future plans and prospects, including
statements regarding the development of luspatercept and
Acceleron’s TGF-beta superfamily program generally, the timeline
for clinical development and regulatory approval of luspatercept,
the expected timing for the reporting of data from ongoing trials,
and the structure of Acceleron’s planned or pending clinical
trials. Each forward-looking statement is subject to risks and
uncertainties that could cause actual results to differ materially
from those expressed or implied in such statement. Applicable risks
and uncertainties include that preclinical testing of Acceleron’s
compounds and data from clinical trials may not be predictive of
the results or success of ongoing or later clinical trials, that
data may not be available when we expect it to be, that Acceleron
or Celgene will be unable to successfully complete the clinical
development of its compounds, that the development of Acceleron's
compounds will take longer or cost more than planned, that
Acceleron or Celgene may be delayed in initiating or completing any
clinical trials, and that Acceleron's compounds will not receive
regulatory approval or become commercially successful products.
Other risks and uncertainties include those identified under the
heading "Risk Factors" included in Acceleron's Annual Report on
Form 10-K which was filed with the Securities and Exchange
Commission (SEC) on March 2, 2015, and other filings that Acceleron
has made and may make with the SEC in the future. The
forward-looking statements contained in this press release reflect
Acceleron’s current views with respect to future events, and
Acceleron does not undertake and specifically disclaims any
obligation to update any forward-looking statements.
Celgene:
This press release contains forward-looking statements, which
are generally statements that are not historical facts.
Forward-looking statements can be identified by the words
"expects," "anticipates," "believes," "intends," "estimates,"
"plans," "will," “outlook” and similar expressions. Forward-looking
statements are based on management’s current plans, estimates,
assumptions and projections, and speak only as of the date they are
made. We undertake no obligation to update any forward-looking
statement in light of new information or future events, except as
otherwise required by law. Forward-looking statements involve
inherent risks and uncertainties, most of which are difficult to
predict and are generally beyond our control. Actual results or
outcomes may differ materially from those implied by the
forward-looking statements as a result of the impact of a number of
factors, many of which are discussed in more detail in our Annual
Report on Form 10-K and our other reports filed with the Securities
and Exchange Commission.
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Acceleron:Investors:Kevin McLaughlin, 617-649-9204Chief
Financial OfficerorMedia:Suda Communications LLCMaureen L. Suda,
585-387-9248orCelgene:Investors:908-673-9628investors@celgene.comorMedia:908-673-2275media@celgene.com
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