Price of ZYNTEGLO reflects potentially curative
clinical benefit through achievement of durable transfusion
independence and normal or near normal total hemoglobin levels
Innovative outcomes-based contract offering
includes single upfront payment and up to 80% risk-sharing
ZYNTEGLO will be available through a network of
Qualified Treatment Centers with experience delivering novel cell
and gene therapies
“my bluebird support” patient services program
will provide comprehensive, personalized support for patients and
their families
Following the FDA approval of ZYNTEGLO® (betibeglogene
autotemcel), also known as beti-cel, for the treatment of
beta-thalassemia in adult and pediatric patients who require
regular red blood cell (RBC) transfusions, today bluebird bio
released details of its U.S. commercial infrastructure to support
rapid access to ZYNTEGLO, including an innovative, outcomes-based
contract offering and a comprehensive patient support program.
“As the first and only FDA-approved gene therapy for people with
beta-thalassemia who require regular red blood cell transfusions,
and with the potential to untether patients from a lifelong,
burdensome treatment regimen, ZYNTEGLO is setting the standard for
what a one-time therapy can deliver,” said Tom Klima, chief
commercial and operating officer, bluebird bio. “We have developed
an equally innovative commercial infrastructure in collaboration
with payers and providers, and with a focus on patients, to enable
broad and timely access to ZYNTEGLO for eligible patients and to
support patients and families at every step of the treatment
journey.”
Under the current standard of care, patients with
transfusion-dependent beta-thalassemia require RBC transfusions
every two to five weeks and may lose decades of life relative to
the general population. The lifetime cost of medical care for a
patient with transfusion-dependent beta-thalassemia can reach up to
$6.4 million in the U.S. and the average total health care cost per
patient per year is 23 times higher than the general population.
bluebird estimates that there are approximately 1,300-1,500
individuals with transfusion-dependent beta-thalassemia in the
U.S.
bluebird has set the wholesale acquisition cost of ZYNTEGLO in
the U.S. at $2.8M in recognition of its robust and sustained
clinical benefit demonstrated in clinical studies and its potential
to alleviate a lifetime of health care costs associated with
regular RBC transfusions and iron management.
Payers Prepared to Support Access, Reimbursement
bluebird has devised an innovative strategy to enable timely and
quality access to ZYNTEGLO, informed by payer insights and is
committed to flexibility to meet payer needs. This includes one
upfront payment that can be paired with an outcomes-based
agreement. As part of this agreement, bluebird will reimburse
contracted commercial and government payers up to 80% of the cost
of the therapy if a patient fails to achieve and maintain
transfusion independence up to two years following infusion. All
patients in ZYNTEGLO Phase 3 studies who achieved transfusion
independence (TI) have remained transfusion free. This outcomes
measure is recognized by payers and providers as clinically
meaningful and straightforward to track through claims data.
“A one-time therapy with the potential to free patients from
chronic transfusions may change the lives of countless patients and
their families and transform the treatment paradigm for
transfusion-dependent beta-thalassemia,” said Michael Sherman, MD,
MBA, MS, executive vice president and chief medical officer of
Point32Health. “Point32Health is thrilled to collaborate with
bluebird bio to provide access to this first-of-its kind therapy
for our members who currently spend hundreds of hours each year
managing their disease. bluebird’s commitment to refunding up to 80
percent of the treatment cost meaningfully reduces the risk
associated with an upfront payment, and will enable implementation
of an outcomes-based agreement at a scale and magnitude not
previously seen in the U.S.”
Payers have expressed a deep understanding of the unmet need in
transfusion-dependent beta-thalassemia, and the value of ZYNTEGLO.
Approximately 70-75% of patients with transfusion-dependent
beta-thalassemia are covered by commercial insurance, and bluebird
is currently in late-stage negotiations with leading commercial
payers including national Pharmacy Benefit Managers (PBMs) with the
potential to represent dozens of plans. Additionally, we are
engaging with state Medicaid agencies representing approximately
80% of publicly insured thalassemia patients.
Qualified Treatment Centers are Preparing for
Patients
ZYNTEGLO will be available exclusively through bluebird
Qualified Treatment Centers (QTCs)—leading healthcare institutions
selected on the basis of clinical expertise in beta-thalassemia and
other hemoglobinopathies, stem cell transplant, and cell and gene
therapy—to best support the specialized nature of ex-vivo
lentiviral vector gene therapy.
“Our network of Qualified Treatment Centers includes preeminent
transplant institutions from across the U.S. and is committed to
transforming the treatment of patients with beta-thalassemia who
require regular red blood cell transfusions,” said Richard Colvin,
MD, chief medical officer, bluebird bio. “These Centers are
critical partners in our mission for patients and were selected not
only for their exceptional clinical expertise in treating patients
with beta-thalassemia, but for their practical experience with the
specialized manufacturing and novel reimbursement associated with
one-time therapies.”
The ZYNTEGLO QTC network includes established transplant centers
from across the U.S. bluebird plans to onboard QTCs on a rolling
basis, with the first wave of QTCs trained and activated in
September 2022, in anticipation of initiating first patient
apheresis in the fourth quarter.
More information on the ZYNTEGLO Qualified Treatment Center
network will be available through my bluebird support.
my bluebird support Available to Assist Patients and Families
at All Stages of the ZYNTEGLO Treatment Journey
bluebird’s patient support program, my bluebird support, will
offer personalized support for patients and their families related
to all aspects of the gene therapy journey—from education and
resources to support informed decision-making to benefits
verification and logistical support.
Patients and families interested in more information about
ZYNTEGLO or who are beginning the treatment journey can contact my
bluebird support to connect with an experienced Patient Navigator
who can assist with:
- Education on the bluebird bio treatment process, how to
find a Qualified Treatment Center, and questions about insurance
and treatment planning
- Insurance questions including benefits verification,
claims support, and co-pay assistance or other resources for
eligible patients
- Treatment support at all phases of the gene therapy
journey including logistical and financial support for eligible
patients
“We recognize the decision to pursue gene therapy is complex and
deeply personal, and that the treatment process can be a major
undertaking for families. Our goal with my bluebird support is to
provide comprehensive support services that are as customized as
our therapies to enable a seamless experience for patients and
their families,” said Klima.
Patients and caregivers can call 833-888-NEST (833-888-6378)
Monday-Friday between 8 a.m. and 8 p.m. Eastern to ask questions
and enroll. More information will be available at
mybluebirdsupport.com in the coming days.
ZYNTEGLO Clinical Data
bluebird bio has the longest and most robust clinical program in
transfusion-dependent beta‑thalassemia (TDT) in the field of gene
therapy. The approval of ZYNTEGLO is based on data from bluebird
bio’s Phase 3 studies HGB-207 (Northstar-2) and HGB-212
(Northstar-3), and the long-term follow-up study LTF-303.
The single-arm, open-label, 24-month Phase 3 studies of ZYNTEGLO
included 41 patients aged 4 to 34 years with both non-β0/β0 and
β0/β0 genotypes, with longest follow up out to 4 years. Eighty-nine
percent (32/36) of evaluable patients across ages and genotypes
achieved transfusion independence (TI), which is defined as no
longer needing RBC transfusions for at least 12 months while
maintaining a weighted average total hemoglobin of at least 9 g/dL.
Results in these patients were durable as of last follow-up.
The most common non-laboratory adverse reactions (≥20%) were
mucositis, febrile neutropenia, vomiting, pyrexia, alopecia,
epistaxis, abdominal pain, musculoskeletal pain, cough, headache,
diarrhea, rash, constipation, nausea, decreased appetite,
pigmentation disorder, and pruritus. The most common Grade 3 or 4
laboratory abnormalities (>50%) include neutropenia,
thrombocytopenia, leukopenia, anemia, and lymphopenia.
Enrollment is complete and all patients have been treated in the
Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies
evaluating ZYNTEGLO. Follow-up in HGB-212 is ongoing. bluebird bio
is also conducting a long-term follow-up study, LTF-303, to monitor
safety and efficacy for patients with TDT who have participated in
bluebird bio-sponsored clinical studies of lentiviral vector (LVV)
gene therapy through 15 years post-treatment.
Across all studies, all patients who achieved transfusion
independence have remained transfusion-free.
About ZYNTEGLO® (betibeglogene autotemcel) or
beti-cel
ZYNTEGLO is a first-in-class, one-time ex-vivo LVV gene therapy
approved for the treatment of beta-thalassemia in adult and
pediatric patients who require regular red blood cell transfusions.
ZYNTEGLO works by adding functional copies of a modified form of
the beta-globin gene (βA-T87Q-globin gene) into a patient’s own
hematopoietic (blood) stem cells to enable the production of a
modified functional adult hemoglobin (HbAT87Q). Once a patient has
the βA-T87Q-globin gene, they have the potential to increase
ZYNTEGLO-derived adult hemoglobin (HbAT87Q) and total hemoglobin to
normal or near normal levels that can eliminate the need for
regular red blood cell (RBC) transfusions.
Indication
ZYNTEGLO is indicated for the treatment of adult and pediatric
patients with beta-thalassemia who require regular red blood cell
(RBC) transfusions.
Important Safety Information
Delayed Platelet Engraftment
Delayed platelet engraftment has been observed with ZYNTEGLO
treatment. Bleeding risk is increased prior to platelet engraftment
and may continue after engraftment in patients with prolonged
thrombocytopenia; 15% of patients had ≥ Grade 3 decreased platelets
on or after Day 100.
Patients should be made aware of the risk of bleeding until
platelet recovery has been achieved. Monitor patients for
thrombocytopenia and bleeding according to standard guidelines.
Conduct frequent platelet counts until platelet engraftment and
platelet recovery are achieved. Perform blood cell count
determination and other appropriate testing whenever clinical
symptoms suggestive of bleeding arise.
Risk of Neutrophil Engraftment Failure
There is a potential risk of neutrophil engraftment failure
after treatment with ZYNTEGLO. Neutrophil engraftment failure is
defined as failure to achieve three consecutive absolute neutrophil
counts (ANC) ≥ 500 cells/microliter obtained on different days by
Day 43 after infusion of ZYNTEGLO. Monitor neutrophil counts until
engraftment has been achieved. If neutrophil engraftment failure
occurs in a patient treated with ZYNTEGLO, provide rescue treatment
with the back-up collection of CD34+ cells.
Risk of Insertional Oncogenesis
There is a potential risk of LVV mediated insertional
oncogenesis after treatment with ZYNTEGLO.
Patients treated with ZYNTEGLO may develop hematologic
malignancies and should be monitored lifelong. Monitor for
hematologic malignancies with a complete blood count (with
differential) at Month 6 and Month 12 and then at least annually
for at least 15 years after treatment with ZYNTEGLO, and
integration site analysis at Months 6, 12, and as warranted.
In the event that a malignancy occurs, contact bluebird bio at 1
833-999-6378 for reporting and to obtain instructions on collection
of samples for testing.
Hypersensitivity Reactions
Allergic reactions may occur with the infusion of ZYNTEGLO. The
dimethyl sulfoxide (DMSO) in ZYNTEGLO may cause hypersensitivity
reactions, including anaphylaxis.
Anti-retroviral and Hydroxyurea Use
Patients should not take prophylactic HIV anti-retroviral
medications or hydroxyurea for at least one month prior to
mobilization, or for the expected duration for elimination of the
medications, and until all cycles of apheresis are completed. If a
patient requires anti-retrovirals for HIV prophylaxis, then confirm
a negative test for HIV before beginning mobilization and apheresis
of CD34+ cells.
Interference with Serology Testing
Patients who have received ZYNTEGLO are likely to test positive
by polymerase chain reaction (PCR) assays for HIV due to integrated
BB305 LVV proviral DNA, resulting in a false-positive test for HIV.
Therefore, patients who have received ZYNTEGLO should not be
screened for HIV infection using a PCR‑based assay.
Adverse Reactions
The most common non-laboratory adverse reactions (≥20%) were
mucositis, febrile neutropenia, vomiting, pyrexia, alopecia,
epistaxis, abdominal pain, musculoskeletal pain, cough, headache,
diarrhea, rash, constipation, nausea, decreased appetite,
pigmentation disorder, and pruritus. The most common Grade 3 or 4
laboratory abnormalities (>50%) include neutropenia,
thrombocytopenia, leukopenia, anemia, and lymphopenia.
Drug Interactions
Drug-drug interactions between iron chelators and the
myeloablative conditioning agent must be considered. Iron chelators
should be discontinued at least 7 days prior to initiation of
conditioning. The prescribing information for the iron chelator(s)
and the myeloablative conditioning agent should be consulted for
the recommendations regarding co-administration with CYP3A
substrates.
Some iron chelators are myelosuppressive. After ZYNTEGLO
infusion, avoid use of these iron chelators for 6 months. If iron
chelation is needed, consider administration of
non-myelosuppressive iron chelators. Phlebotomy can be used in lieu
of iron chelation, when appropriate.
Pregnancy/Lactation
Advise patients of the risks associated with conditioning
agents, including on pregnancy and fertility. ZYNTEGLO should not
be administered to women who are pregnant, and pregnancy after
ZYNTEGLO infusion should be discussed with the treating
physician.
ZYNTEGLO is not recommended for women who are breastfeeding, and
breastfeeding after ZYNTEGLO infusion should be discussed with the
treating physician.
Females and Males of Reproductive Potential
A negative serum pregnancy test must be confirmed prior to the
start of mobilization and re-confirmed prior to conditioning
procedures and before ZYNTEGLO administration.
Women of childbearing potential and men capable of fathering a
child should use an effective method of contraception (intra
uterine device or combination of hormonal and barrier
contraception) from start of mobilization through at least 6 months
after administration of ZYNTEGLO.
Advise patients of the option to cryopreserve semen or ova
before treatment if appropriate.
Please see full Prescribing Information for ZYNTEGLO.
About bluebird bio, Inc.
bluebird bio is pursuing curative gene therapies to give
patients and their families more bluebird days. With a dedicated
focus on severe genetic diseases, bluebird has industry-leading
clinical and research programs for sickle cell disease,
beta-thalassemia and cerebral adrenoleukodystrophy and is advancing
research to apply new technologies to these and other diseases. We
custom design each of our therapies to address the underlying cause
of disease and have developed in-depth and effective analytical
methods to understand the safety of our lentiviral vector
technologies and drive the field of gene therapy forward.
Founded in 2010, bluebird has the largest and deepest ex-vivo
gene therapy data set in the world—setting the standard for
industry. Today, bluebird continues to forge new paths, combining
our real-world experience with a deep commitment to patient
communities and a people-centric culture that attracts and grows a
diverse flock of dedicated birds.
For more information, visit bluebirdbio.com or follow us
on social media at @bluebirdbio, LinkedIn,
Instagram and YouTube.
ZYNTEGLO and bluebird bio are trademarks of bluebird bio,
Inc.
bluebird bio Cautionary Statement Regarding Forward-Looking
Statements
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of
1995. All statements that are not statements of historical facts
are, or may be deemed to be, forward-looking statements, such as
statements regarding the commercialization of ZYNTEGLO, including
without limitation the likelihood of executing binding agreements
with payers, our expectations on timing for activating QTCs, our
expectations on the timing and size for expanding our QTC network,
our plans for the first patient apheresed in the commercial
setting, and the availability of services offered by my bluebird
support program to support patient treatment. Such forward-looking
statements are based on historical performance and current
expectations and projections about our future goals, plans and
objectives and involve inherent risks, assumptions and
uncertainties, including internal or external factors that could
delay, divert or change any of them in the next several years, that
are difficult to predict, may be beyond our control and could cause
our future goals, plans and objectives to differ materially from
those expressed in, or implied by, the statements. No
forward-looking statement can be guaranteed. Forward-looking
statements in this press release should be evaluated together with
the many risks and uncertainties that affect bluebird bio’s
business, particularly those identified in the risk factors
discussion in bluebird bio’s Annual Report on Form 10-K, as updated
by our subsequent Quarterly Reports on Form 10-Q, Current Reports
on Form 8-K and other filings with the Securities and Exchange
Commission. These risks and uncertainties include, but are not
limited to: the risk that the efficacy and safety results from our
prior and ongoing clinical trials will not continue or be seen in
the commercial context, including that patients do not achieve or
maintain transfusion independence; the risk that we are not able to
activate QTCs on the timeframe that we expect; the risk that the
QTCs experience delays in their ability to enroll or treat
patients; the risk that we experience delays in establishing
operational readiness across our supply chain following approval to
support treatment in the commercial context; the risk that there is
not sufficient patient demand or payer reimbursement to support
continued commercialization of ZYNTEGLO; the risk that additional
insertional oncogenic or other safety events associated with
lentiviral vector, drug product, or myeloablation will be
discovered or reported over time; and the risk that eli-cel or
lovo-cel will not be successfully developed, approved by the FDA or
commercialized. The forward-looking statements included in this
document are made only as of the date of this document and except
as otherwise required by applicable law, bluebird bio undertakes no
obligation to publicly update or revise any forward-looking
statement, whether as a result of new information, future events,
changed circumstances or otherwise.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220817005677/en/
Investors: Courtney O’Leary, 978-621-7347
coleary@bluebirdbio.com Media: Jess Rowlands,
857-299-6103 jess.rowlands@bluebirdbio.com
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