U.S. Food and Drug Administration grants
priority review for beta-thalassemia indication and sets target
action date of December 4, 2019
U.S. Food and Drug Administration sets target
action date of April 4, 2020 for myelodysplastic syndromes
indication
Luspatercept EU Marketing Authorization
Application also validated
Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc.
(NASDAQ: XLRN) today announced that the U.S. Food and Drug
Administration (FDA) has accepted Celgene’s Biologics License
Application (BLA) for luspatercept, an investigational erythroid
maturation agent, for the treatment of adult patients with very low
to intermediate-risk myelodysplastic syndromes (MDS)-associated
anemia who have ring sideroblasts and require red blood cell (RBC)
transfusions, and for the treatment of adult patients with
beta-thalassemia-associated anemia who require RBC transfusions.
The FDA has granted Priority Review to this BLA for the evaluation
of the beta-thalassemia indication and set a Prescription Drug User
Fee Act (PDUFA), or target action, date of December 4, 2019. The
FDA has also set a PDUFA date of April 4, 2020 for the evaluation
of the MDS indication.
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“The acceptance of the luspatercept filings and granting of the
U.S. priority review for beta-thalassemia represent another
important step in delivering this novel therapy to patients in
need,” said Jay Backstrom, M.D., Chief Medical Officer for Celgene.
“We believe that luspatercept can play a critical role in treating
the anemia associated with these serious blood diseases, and with
these milestones achieved we look forward to working closely with
the agency to move this therapy toward approval.”
The safety and efficacy results provided in the application are
from the pivotal phase 3 studies MEDALIST and BELIEVE, which
evaluated the ability of luspatercept to effectively treat the
anemia associated with MDS and beta-thalassemia, respectively.
MEDALIST and BELIEVE were both presented at the 2018 American
Society of Hematology annual meeting, where MEDALIST was included
in the plenary session.
The companies also announced that Celgene’s Marketing
Authorization Application in the EU has been successfully validated
and the review is now underway.
“The ongoing U.S. and European regulatory reviews of the
luspatercept filings in MDS and beta-thalassemia strongly support
our primary goal, which has always been to bring a potentially
transformative new treatment to these patients with unmet clinical
need as quickly as possible,” said Habib Dable, President and Chief
Executive Officer of Acceleron. “At the same time, we continue to
explore the ability of luspatercept to address anemia in additional
settings, including patients with treatment-naïve MDS,
non-transfusion-dependent beta-thalassemia, and myelofibrosis.”
Luspatercept is an investigational therapy that is not approved
for any use in any country.
About Luspatercept
Luspatercept is a first-in-class erythroid maturation agent
(EMA) that regulates late-stage red blood cell maturation.
Acceleron and Celgene are jointly developing luspatercept
as part of a global collaboration. A phase 3 trial (COMMANDS) in
ESA-naïve, lower-risk MDS patients, the BEYOND phase 2 trial in
non-transfusion-dependent beta-thalassemia, and a phase 2 trial in
myelofibrosis are ongoing. For more information, please
visit www.clinicaltrials.gov.
About MEDALIST
MEDALIST is a phase 3, randomized, double blind,
placebo-controlled, multi-center study evaluating the safety and
efficacy of luspatercept in adults with very low-, low-, or
intermediate-risk myelodysplastic syndromes (MDS). All patients
were RBC transfusion dependent and were either refractory or
intolerant to prior erythropoiesis-stimulating agent (ESA) therapy
or were ESA naïve with endogenous serum erythropoietin ≥ 200 U/L
and had no prior treatment with disease modifying agents. The
median age of the patients enrolled in the trial was 71 years in
the luspatercept treatment group and 72 years in the placebo group.
Median transfusion burden in both treatment arms was 5 RBC units/8
weeks. 229 patients were randomized to receive either luspatercept
1.0 mg/kg (153 patients) or placebo (76 patients) by subcutaneous
injection once every 21 days. The study was conducted at 65 sites
in 11 countries.
About BELIEVE
BELIEVE is a phase 3, randomized, double blind,
placebo-controlled multicenter study comparing luspatercept + best
supportive care (BSC) versus placebo + BSC in adults with
beta-thalassemia patients who require regular RBC transfusions. The
median age of the patients was 30 years in both treatment arms. 336
patients were randomized to receive either luspatercept 1.0 mg/kg
(224 patients) or placebo (112 patients) by subcutaneous injection
every 21 days for up to 48 weeks. Crossover to the luspatercept
treatment groups was allowed after unblinding based on the
recommendation of an independent Data Safety Monitoring Committee;
patients treated with luspatercept will be followed for up to 3
years. The study was conducted at 65 sites in 15 countries.
About Celgene
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 next-generation solutions in protein homeostasis,
immuno-oncology, epigenetics, immunology and neuro-inflammation.
For more information, please visit www.celgene.com.
Follow Celgene on Social Media: Twitter, Pinterest, LinkedIn,
Facebook and YouTube.
About Acceleron
Acceleron is a clinical-stage biopharmaceutical company
dedicated to the discovery, development, and commercialization of
therapeutics to treat serious and rare diseases. The Company's
leadership in the understanding of TGF-beta superfamily biology and
protein engineering generates innovative compounds that engage the
body's ability to regulate cellular growth and repair.
Acceleron focuses its research and development efforts in
hematologic, neuromuscular, and pulmonary diseases. In hematology,
the Company and its global collaboration partner, Celgene, are
developing luspatercept for the treatment of chronic anemia in
myelodysplastic syndromes, beta-thalassemia, and myelofibrosis.
Acceleron is also advancing its neuromuscular program with ACE-083,
a locally-acting Myostatin+ agent in Phase 2 development in
facioscapulohumeral muscular dystrophy and Charcot-Marie-Tooth
disease and is conducting a Phase 2 pulmonary program with
sotatercept in pulmonary arterial hypertension.
For more information, please visit www.acceleronpharma.com.
Follow Acceleron on Social
Media: @AcceleronPharma and LinkedIn.
FORWARD-LOOKING STATEMENTS
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995. Such forward-looking statements include those regarding the
potential benefits of, and plans relating to the collaboration
between Acceleron and Celgene; the potential of luspatercept
as a therapeutic drug; and the benefit of each company’s strategic
plans and focus. The words “anticipate,” “believe,” “estimate,”
“expect,” “intend,” “may,” “plan,” “predict,” “project,” “will,”
“would,” “could,” “potential,” “possible,” “hope” and similar
expressions are intended to identify forward-looking statements,
although not all forward-looking statements contain these
identifying words. Such statements are subject to numerous
important factors, risks and uncertainties that may cause actual
events or results to differ materially from current expectations
and beliefs. For example, there can be no guarantee that
luspatercept will be successfully developed or complete necessary
clinical phases. Forward-looking statements in this press release
could also be affected by risks and uncertainties relating to a
number of other important factors, including: results of clinical
trials, including subsequent analysis of existing data and new data
received from ongoing and future studies; the content and timing of
decisions made by the U.S. FDA and other regulatory
authorities, investigational review boards at clinical trial sites
and publication review bodies; the ability to obtain and maintain
requisite regulatory approvals and to enroll patients in planned
clinical trials; the ability to obtain, maintain and enforce patent
and other intellectual property protection for luspatercept; the
ability to maintain key collaborations; and general economic and
market conditions. These and other risks are described in greater
detail under the caption "Risk Factors" included in each company’s
public filings with the Securities and Exchange Commission and
with respect to Celgene includes risk factors related to the
proposed transaction between Bristol-Myers Squibb and Celgene, such
as, but not limited to, the risks that: management’s time and
attention is diverted on transaction related issues; disruption
from the transaction makes it more difficult to maintain business,
contractual and operational relationships; and Bristol-Myers
Squibb, Celgene or the combined company is unable to retain key
personnel. Any forward-looking statements contained in this press
release speak only as of the date hereof, and neither company has
any obligation to update any forward-looking statements, whether as
a result of new information, future events or otherwise, except as
may be required by law.
Hyperlinks are provided as a convenience and for informational
purposes only. Neither Celgene nor Acceleron bears
responsibility for the security or content of external websites or
websites outside of their respective control.
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Celgene
CorporationInvestors:+1-908-673-9628ir@celgene.comorMedia:+1-908-673-2275media@celgene.comAcceleron
Pharma Inc.Investors:Todd James, IRC, (617) 649-9393Vice
President, Investor Relations and Corporate
CommunicationsorMedia:Matt Fearer, (617) 301-9557Director,
Corporate Communications
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