REBLOZYL is the first and only FDA-approved
erythroid maturation agent, representing a new class of therapy for
these patients
Approval of REBLOZYL marks the first
FDA-approved treatment for anemia in beta thalassemia
Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc.
(NASDAQ: XLRN) today announced the U.S. Food and Drug
Administration (FDA) has approved REBLOZYL® (luspatercept-aamt) for
the treatment of anemia in adult patients with beta thalassemia who
require regular red blood cell (RBC) transfusions.1 REBLOZYL is not
indicated for use as a substitute for RBC transfusions in patients
who require immediate correction of anemia.1 REBLOZYL is the first
and only FDA-approved erythroid maturation agent, representing a
new class of therapy which works by regulating late-stage red blood
cell maturation to help patients reduce their RBC transfusion
burden.1
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“Today’s approval is an important milestone and underscores our
continued commitment to patients with hematology disorders,” said
Nadim Ahmed, President, Global Hematology and Oncology for Celgene.
“There are very limited options for patients living with anemia due
to beta thalassemia who are dependent on long term red blood cell
transfusions. We are pleased to make REBLOZYL available as a new
therapy for these patients to help address their anemia, a
significant clinical complication of beta thalassemia.”2
“We’re thrilled that Acceleron’s first approved medicine is one
with the potential to help patients with beta thalassemia, who have
been in need of new treatments for this lifelong disease,” said
Habib Dable, President and Chief Executive Officer of Acceleron.
“We are enormously grateful to the patients, families and
caregivers who participated in and supported our research. Their
contributions have been essential in helping to ensure that
REBLOZYL would emerge successfully from our longstanding
collaboration with Celgene.”
Beta thalassemia is a rare, inherited blood disorder caused by a
genetic defect in hemoglobin. The disease is associated with
ineffective erythropoiesis, which results in the production of
fewer and less healthy RBCs, often leading to severe anemia – a
condition that can be debilitating and can lead to more severe
complications for patients – as well as other serious health
issues.2,3 Treatment options for anemia associated with beta
thalassemia are limited, consisting mainly of RBC transfusions,
which have the potential to contribute to iron overload, which can
cause serious complications such as organ damage.4
The approval of REBLOZYL for beta thalassemia, which received a
Priority Review designation from the FDA, is based on results from
the pivotal, Phase 3, randomized, double-blind, placebo-controlled,
multicenter BELIEVE trial evaluating the safety and efficacy of
REBLOZYL for the treatment of anemia in adult patients with beta
thalassemia who require regular RBC transfusions (defined as 6-20
RBC units per 24 weeks, with no transfusion-free period greater
than 35 days during that period).1 All patients were eligible to
receive best supportive care, which included RBC transfusions;
iron-chelating agents; use of antibiotic, antiviral, and antifungal
therapy; and/or nutritional support, as needed.1 The trial achieved
a clinically meaningful and statistically significant improvement
in the primary endpoint.1 In the REBLOZYL arm, 21.4% of patients
(n=48) achieved a ≥33% reduction from baseline in RBC transfusion
burden (with a reduction of at least 2 units) during weeks 13–24
after randomization, compared to 4.5% (n=5) in the placebo arm
(risk difference [95% CI]: 17.0 [10.4, 23.6], P<0.0001).1
The study also met key secondary endpoints, including
transfusion burden reduction of at least 33% (with a reduction of
at least 2 units), during weeks 37 to week 48, which was achieved
in 19.6% (n=44) of patients in the REBLOZYL arm and 3.6% (n=4) in
the placebo arm (risk difference [95% CI]: 16.1 [9.8, 22.4],
P<0.0001).1
Other efficacy endpoints included transfusion burden reduction
of ≥50% (with a reduction of at least 2 units) during weeks 13-24
and weeks 37-48.1 A ≥50% reduction in transfusion burden was
observed in 7.6% of patients (n=17) receiving REBLOZYL vs. 1.8% of
patients (n=2) in the placebo arm at weeks 13-24 (risk difference
[95% CI]: 5.8 [1.6, 10.1], P=0.0303), and 10.3% of patients (n=23)
vs. 0.9% of patients (n=1) at weeks 37-48 (risk difference [95%
CI]: 9.4 [5, 13.7], P=0.0017), respectively.1
In the BELIEVE trial,
thromboembolic events, including deep vein thromboses, pulmonary
embolus, portal vein thrombosis, and ischemic stroke, were
experienced in 3.6% (8/223) of REBLOZYL treated patients.1
Hypertension was reported in 10.7% (61/571) of REBLOZYL-treated
patients across the clinical development program.1 REBLOZYL may
cause fetal harm when administered to a pregnant woman.
Serious adverse reactions
occurred in 3.6% of patients receiving REBLOZYL.1 Serious adverse
reactions reported in 1% of patients were cerebrovascular accident
and deep vein thrombosis.1 One patient died due to an unconfirmed
case of AML.1 The most common adverse reactions (at least 10% for
REBLOZYL, and 1% more than placebo) were headache (26% vs 24%),
bone pain (20% vs 8%), arthralgia (19% vs 12%), fatigue (14% vs
13%), cough (14% vs 11%), abdominal pain (14% vs 12%), diarrhea
(12% vs 10%) and dizziness (11% vs 5%).1
Permanent discontinuation due
to an adverse reaction (Grades 1-4) occurred in 5.4% of patients
who received REBLOZYL.1 The most frequent adverse reactions
requiring permanent discontinuation in patients who received
REBLOZYL included arthralgia (1%), back pain (1%), bone pain
(<1%), and headache (<1%).1 Dosage reductions due to an
adverse reaction occurred in 2.7% of patients who received
REBLOZYL.1 The most frequent adverse reactions requiring a dosage
reduction in >0.5% of patients who received REBLOZYL included
hypertension and headache.1 Dosage interruptions due to an adverse
reaction occurred in 15.2% of patients who received REBLOZYL.1 The
most frequent adverse reactions requiring a dosage interruption in
>1% of patients who received REBLOZYL included upper respiratory
tract infection, ALT increase, and cough.1
REBLOZYL is anticipated to be
available 1 week following the FDA approval.
About BELIEVE BELIEVE is a Phase 3, randomized,
double-blind, placebo-controlled multicenter study comparing
REBLOZYL + best supportive care (BSC) versus placebo + BSC in
adults who require regular RBC transfusions (6-20 RBC units per 24
weeks with no transfusion-free period greater than 35 days during
that period) due to beta thalassemia.1 Best supportive care
included RBC transfusions; iron-chelating agents; use of
antibiotic, antiviral, and antifungal therapy; and/or nutritional
support as needed. The median age of patients was 30 years in both
treatment arms.1 336 patients were randomized 2:1 to receive either
REBLOZYL (224 patients) or placebo (112 patients) at a starting
dose of 1.0 mg/kg by subcutaneous injection every 21 days for up to
48 weeks.1 Patients had the option to cross over to the
luspatercept-aamt treatment groups only after all patients
completed the treatment period, and after unblinding based on the
recommendation of an independent Data Safety Monitoring Committee.
Patients treated with luspatercept-aamt will be followed for up to
5 years. The study was conducted at 65 sites in 15 countries.1
About REBLOZYL® REBLOZYL is a first-in-class erythroid
maturation agent that promotes late-stage red blood cell maturation
in animal models.1 Celgene and Acceleron are jointly developing
REBLOZYL as part of a global collaboration.
Additional Clinical Investigation
The FDA is also evaluating luspatercept-aamt for the treatment of
anemia in adults with very low- to intermediate-risk
myelodysplastic syndromes (MDS) who have ring sideroblasts and
require RBC transfusions. The FDA has set a Prescription Drug User
Fee Act (PDUFA), or target action, date of April 4, 2020 for this
indication. In Europe, Celgene’s Marketing Authorization
Application for the treatment of anemia in adults with beta
thalassemia or MDS is currently under review.
A Phase 2 trial (BEYOND) in adult patients with
non-transfusion-dependent beta thalassemia5; a Phase 3 trial
(COMMANDS) in ESA-naïve, lower-risk MDS patients6; and a Phase 2
trial in myelofibrosis patients are ongoing.7 For more information,
please visit www.clinicaltrials.gov.
REBLOZYL has not been approved as safe and effective for use in
patients with MDS or myelofibrosis in any country.
Indication REBLOZYL is indicated for the treatment of anemia
in adult patients with beta thalassemia who require regular red
blood cell (RBC) transfusions
REBLOZYL is not indicated for use as a substitute for RBC
transfusions in patients who require immediate correction of
anemia
Important Safety Information
WARNINGS AND PRECAUTIONS Thrombosis/Thromboembolism
Thromboembolic events (TEE) were reported in 8/223 (3.6%)
REBLOZYL-treated patients. TEEs included deep vein thrombosis,
pulmonary embolus, portal vein thrombosis, and ischemic stroke.
Patients with known risk factors for thromboembolism, (splenectomy
or concomitant use of hormone replacement therapy), may be at
further increased risk of thromboembolic conditions. Consider
thromboprophylaxis in patients at increased risk of TEE. Monitor
patients for signs and symptoms of thromboembolic events and
institute treatment promptly.
Hypertension Hypertension was reported in 10.7% (61/571)
of REBLOZYL-treated patients. Across clinical studies, the
incidence of grade 3-4 hypertension ranged from 1.8% to 8.6%. In
patients with beta thalassemia with normal baseline blood pressure,
13 (6.2%) patients developed systolic blood pressure (SBP) >130
mm Hg and 33 (16.6%) patients developed diastolic blood pressure
(DBP) >80 mm Hg. Monitor blood pressure prior to each
administration. Manage new or exacerbations of preexisting
hypertension using anti-hypertensive agents.
Embryo-Fetal Toxicity REBLOZYL may cause fetal harm when
administered to a pregnant woman. REBLOZYL caused increased
post-implantation loss, decreased litter size, and an increased
incidence of skeletal variations in pregnant rat and rabbit
studies. Advise pregnant women of the potential risk to a fetus.
Advise females of reproductive potential to use effective
contraception during treatment and for at least 3 months after the
last dose.
ADVERSE REACTIONS Serious adverse reactions occurring in
1% of patients included cerebrovascular accident and deep vein
thrombosis. A fatal adverse reaction occurred in one patient
treated with REBLOZYL who died due to an unconfirmed case of
AML.
Most common adverse reactions (at least 10% for REBLOZYL, and 1%
more than placebo) were headache (26% vs 24%), bone pain (20% vs
8%), arthralgia (19% vs 12%), fatigue (14% vs 13%), cough (14% vs
11%), abdominal pain (14% vs 12%), diarrhea (12% vs 10%) and
dizziness (11% vs 5%).1
LACTATION It is not known whether REBLOZYL is excreted
into human milk or absorbed systemically after ingestion by a
nursing infant. REBLOZYL was detected in milk of lactating rats.
When a drug is present in animal milk, it is likely that the drug
will be present in human milk. Because many drugs are excreted in
human milk, and because of the unknown effects of REBLOZYL in
infants, a decision should be made whether to discontinue nursing
or to discontinue treatment. Because of the potential for serious
adverse reactions in the breastfed child, breastfeeding is not
recommended during treatment and for 3 months after the last
dose.
Please see full Prescribing Information for REBLOZYL
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 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
co-promoting newly approved REBLOZYL® (luspatercept-aamt) in North
America for the treatment of anemia in adult patients with beta
thalassemia who require regular red blood cell transfusions and
developing luspatercept for the treatment of chronic anemia in
myelodysplastic syndromes and myelofibrosis. Acceleron is also
advancing its neuromuscular program with ACE-083, a locally-acting
Myostatin+ agent in Phase 2 development in 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 REBLOZYL ® (luspatercept-aamt) 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 REBLOZYL will be successfully
commercialized, or continue to be 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 ongoing or
planned clinical trials; the ability to obtain, maintain and
enforce patent and other intellectual property protection for
REBLOZYL; 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.
1 REBLOZYL U.S. Prescribing Information. Accessed November
2019.
2 National Center for Advancing Translational Sciences, Genetic
and Rare Diseases Information Center. Beta-thalassemia. Available
at:
http://rarediseases.info.nih.gov/gard/871/beta-thalassemia/resources/1.
Accessed October 2019.
3 National Institutes of Health: Genetics Home Reference.
Beta-thalassemia. Available at:
https://ghr.nlm.nih.gov/condition/beta-thalassemia#inheritance.
Accessed October 2019.
4 Galanello R and Origa R. Beta-thalassemia. Orphanet J Rare
Dis. 2010 May 21;5:11. Available at:
https://ojrd.biomedcentral.com/articles/10.1186/1750-1172-5-11.
Accessed October 2019.
5 ClinicalTrials.gov. A Study to Determine the Efficacy and
Safety of Luspatercept in Adults With Non Transfusion Dependent
Beta (β)-Thalassemia (BEYOND). Available at:
https://www.clinicaltrials.gov/ct2/show/NCT03342404?term=BEYOND&cond=Beta-Thalassemia&rank=2.
Accessed October 2019.
6 ClinicalTrials.gov. Efficacy and Safety Study of Luspatercept
(ACE-536) Versus Epoetin Alfa for the Treatment of Anemia Due to
IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes
(MDS) in ESA Naïve Subjects Who Require Red Blood Cell Transfusions
(COMMANDS). Available at:
https://www.clinicaltrials.gov/ct2/show/NCT03682536?term=COMMANDS+luspatercept&rank=1.
Accessed October 2019.
7 ClinicalTrials.gov. A Safety and Efficacy Study to Evaluate
Luspatercept in Subjects With Myeloproliferative
Neoplasm-associated Myelofibrosis Who Have Anemia With and Without
Red Blood Cell-transfusion Dependence. Available at:
https://www.clinicaltrials.gov/ct2/show/NCT03194542?term=luspatercept&cond=Myelofibrosis&rank=1.
Accessed October 2019.
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Celgene Corporation Investors: (908) 673-9628
ir@celgene.com or Media: (908) 673-2275 media@celgene.com
Acceleron Pharma Inc. Investors: Todd James, IRC, (617)
649-9393 Vice President, Investor Relations and Corporate
Communications
Ed Joyce, (617) 649-9242 Director, Investor Relations
or Media: Matt Fearer, (617) 301-9557 Director, Corporate
Communications
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