Outcomes-based contract offerings available to
both commercial payers and Medicaid
LYFGENIA will be available through bluebird’s
established national network of Qualified Treatment Centers
beginning in Q1 2024
“my bluebird support” patient services program
will provide personalized support for patients and their families
throughout their treatment journey
Price of LYFGENIA reflects its value as a
potentially curative gene therapy for sickle cell disease through
durable production of anti-sickling adult hemoglobin (HbAT87Q) and
resolution of vaso-occlusive events
bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the
“bluebird”) today announced the details of its U.S. commercial
infrastructure to support timely, equitable access to LYFGENIA™
(lovotibeglogene autotemcel, also known as lovo-cel), an
FDA-approved gene therapy for the treatment of patients 12 years of
age and older with sickle cell disease and a history of
vaso-occlusive events (VOEs). The LYFGENIA launch builds on
bluebird’s demonstrated success delivering ex vivo gene therapies
to patients in the United States and includes outcomes-based
contract offerings for payers, as well as a personalized patient
support program.
“Our approach is built on the principle that timely, equitable
access to gene therapy is imperative for people living with sickle
cell disease,” said Tom Klima, chief commercial & operating
officer, bluebird bio. “As an established commercial gene therapy
leader, we believe bluebird is well positioned to partner with
providers and payers to deliver LYFGENIA to a community that is
long overdue for meaningful treatment advances. The location of our
Qualified Treatment Centers and our patient services offering are
designed to minimize burdens for patients and their families and
caregivers, while our outcomes-based contract offerings have been
created specifically to address challenges payers face as they
adapt to provide access to one-time, transformative therapies.”
Data from clinical studies support LYFGENIA as a potentially
curative gene therapy for sickle cell disease through durable
production of anti-sickling adult hemoglobin (HbAT87Q) and
resolution of vaso-occlusive events. bluebird has set the wholesale
acquisition cost of LYFGENIA in the U.S. at $3.1M in recognition of
the value the therapy may deliver through robust and sustained
clinical benefits and the estimated lifetime impact that reducing
or eliminating VOEs may have on patients’ healthcare utilization,
future earnings, and life opportunities.
The burden of sickle cell disease is immense and can impact
every aspect of life for patients and their loved ones. Beyond the
debilitating and unpredictable vaso-occlusive events that are most
associated with the disease, patients are at risk for irreversible
damage to vital organs, diminished quality of life, and early
death. Fifty to 60% of adults living with sickle cell disease have
end organ damage, and one in four people living with sickle cell
disease have a stroke by age 45. Despite a median age of death of
45 years, it is estimated that U.S. patients with frequent VOEs
average $4.0 -$6.0 million in direct lifetime medical costs, not
including patient-incurred out-of-pocket costs or the impact on
caregivers. Patients also forgo approximately $1.3 million in
lifetime earnings compared to their peers based on how their
disease can limit academic and professional opportunities.
Payers Are Anticipated to Enable Timely Access and
Reimbursement
To support timely and equitable access, bluebird has designed
outcomes-based contract options unique to LYFGENIA that offer
payers meaningful risk sharing tied to VOE-related
hospitalizations—a claims-based metric that is directly correlated
with clinical benefit and aligned with study endpoints in the
LYFGENIA clinical development program—with patients followed for
three years. A second contracting option is available specifically
for state Medicaid agencies, based on direct input from these
payers that predictability and operational ease are essential for
states that are grappling with resource constraints. Both options
tie the cost of LYFGENIA to its performance and manage payer risk
as clinical experience is translated into the commercial
setting.
“The FDA approval of a sickle cell disease gene therapy may be a
game changer,” said Dr. Shantel Herbert-Magee, Chief Medical
Director, Louisiana Medicaid. “Knowing that serious consequences of
sickle cell disease can now potentially be averted, there is an
expectation for Medicaid programs to provide access to cutting-edge
therapies that seemingly have been impenetrable for the
disadvantaged and underinsured. Given the vulnerabilities of the
Medicaid population, we must not only consider the near-term drug
cost but the potential for lower long-term health expenditures.
Hence, policymakers are exploring collective approaches to making
access less challenging from a fiscal and operational stance,
including working with manufacturers on innovative contracts and
examining the geographic and provider barriers to the
administration of gene therapy. Indisputably, Medicaid is critical
to the strategy.”
bluebird is in advanced discussions with the nation’s largest
commercial payers and more than 15 Medicaid agencies representing
80% of individuals with sickle cell disease in the U.S.
Qualified Treatment Centers Are Ready for Patients
LYFGENIA will be available through bluebird’s established
national network of Qualified Treatment Centers (QTCs), leading
healthcare institutions with experience in delivering gene and cell
therapies in the commercial setting, beginning in early 2024. One
hundred percent of those centers have initiated the activation
process for LYFGENIA.
Centers will be onboarded to deliver LYFGENIA on a rolling
basis. Today, 27 QTCs are ready to receive patient referrals and we
anticipate the full network will be fully activated by the end of
Q1 2024. Details are available at
mybluebirdsupport.com/qualified-treatment-center-locator.
my bluebird support Available to Assist Patients and Families
Throughout the LYFGENIA Treatment Journey
bluebird’s patient support program, my bluebird support, will
offer personalized support for each patient/family who enrolls in
the program related to all stages of the gene therapy journey—from
education and decision-making resources to benefits verification
and logistical and financial support for eligible patients.
bluebird’s Patient Navigators are experienced professionals with
success supporting patients with both commercial and public
insurance and are in place now to assist in accessing bluebird’s
FDA-approved therapies. Patients can call 833-888-NEST
(833-888-6378) or visit mybluebirdsupport.com for more
information.
LYFGENIA Clinical Data
The FDA approval of LYFGENIA builds on decades of research into
lentiviral vector gene addition therapy and the largest clinical
development program of any gene therapy for sickle cell
disease.
The label is based on data from patients from the Phase 1/2
HGB-206 study. Safety data supporting the application includes data
from 54 patients who initiated stem cell collection. Efficacy for
LYFGENIA was supported by data from 36 patients in the Phase 1/2
HGB-206 Group C study following enhancements to the treatment and
manufacturing processes made through the course of the clinical
development program. 32 patients were evaluable for the endpoints
of complete resolution of VOEs and severe VOEs in the 6-18 months
post-infusion including 8 adolescent patients. In this cohort:
- Severe vaso-occlusive events were resolved in 30/32 patients
(94%)
- 28/32 patients (88.2%) experienced no vaso-occlusive events at
all
In the studies, VOEs are defined as episodes of acute pain with
no medically determined cause other than a vaso-occlusion, lasting
more than two hours and severe enough to require care at a medical
facility. This includes acute chest syndrome requiring oxygen
treatment and/or blood transfusion, acute hepatic sequestration,
acute priapism lasting 2 hours and requiring care at a medical
facility and acute splenic sequestration. sVOEs require a 24-hour
hospital stay or emergency room visit, or at least two visits to a
hospital or emergency room over a 72-hour period, with both visits
requiring intravenous treatment; all VOEs of priapism are also
considered sVOEs.
The most common adverse reactions ≥ Grade 3 (incidence ≥ 20%)
were stomatitis, thrombocytopenia, neutropenia, febrile
neutropenia, anemia, and leukopenia. As previously reported, three
patients died during LYFGENIA clinical trials; one from sudden
cardiac death due to underlying disease and two from acute myeloid
leukemia who were treated with an earlier version of LYFGENIA using
a different manufacturing process and transplant procedure. Please
see LYFGENIA Important Safety Information below, including a Boxed
Warning for Hematologic Malignancy.
Patients treated with LYFGENIA in bluebird bio-sponsored
clinical studies will be monitored for a total of 15 years through
a long-term safety and efficacy follow-up study (LTF-307).
About LYFGENIA™ LYFGENIA is a one-time ex-vivo lentiviral
vector gene therapy approved for the treatment of patients 12 years
of age or older with sickle cell disease and a history of
vaso-occlusive events (VOEs). LYFGENIA works by adding a functional
β-globin gene to patients’ own hematopoietic (blood) stem cells
(HSCs). Durable production of adult hemoglobin with anti-sickling
properties (HbAT87Q) is possible following successful engraftment.
HbAT87Q has a similar oxygen-binding affinity to wild-type HbA,
limits sickling of red blood cells and has the potential to reduce
and VOEs.
The Phase 1/2 HGB-206 study of LYFGENIA is ongoing with
enrollment and treatment complete; and the Phase 3 HGB-210 study
evaluating LYFGENIA is ongoing. bluebird bio is also conducting a
long-term safety and efficacy follow-up study (LTF-307) for
patients with sickle cell disease who have been treated with
LYFGENIA in bluebird bio-sponsored clinical studies.
Indication LYFGENIA is indicated for the treatment of
patients 12 years of age or older with sickle cell disease and a
history of vaso-occlusive events (VOEs).
Limitations of Use Following
treatment with LYFGENIA, patients with α-thalassemia trait (α3.7/
α3.7) may experience anemia with erythroid dysplasia that may
require chronic red blood cell transfusions. LYFGENIA has not been
studied in patients with more than two α-globin gene deletions.
Important Safety Information
Boxed WARNING: HEMATOLOGIC MALIGNANCY
Hematologic malignancy has occurred in patients treated with
LYFGENIA. Monitor patients closely for evidence of
malignancy through complete blood counts at least every 6 months
and through integration site analysis at Months 6, 12, and
as warranted.
Hematologic Malignancy
Hematologic malignancy has occurred in patients treated with
LYFGENIA (Study 1, Group A). At the time of initial product
approval, two patients treated with an earlier version of LYFGENIA
using a different manufacturing process and transplant procedure
(Study 1, Group A) developed acute myeloid leukemia (AML). One
patient with α-thalassemia trait (Study 1, Group C) has been
diagnosed with myelodysplastic syndrome (MDS).
The additional hematopoietic stress associated with
mobilization, conditioning, and infusion of LYFGENIA, including the
need to regenerate the hematopoietic system, may increase the risk
of a hematologic malignancy. Patients with sickle cell disease have
an increased risk of hematologic malignancy as compared to the
general population.
Patients treated with LYFGENIA may develop hematologic
malignancies and should have lifelong monitoring. Monitor for
hematologic malignancies with a complete blood count (with
differential) at least every 6 months for at least 15 years after
treatment with LYFGENIA, 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.
Post-Marketing Long Term Follow-Up
Study: Patients who intend to receive treatment with
LYFGENIA are encouraged to enroll in the study, as available, to
assess the long-term safety of LYFGENIA and the risk of
malignancies occurring after treatment with LYFGENIA by calling
bluebird bio at 1-833-999-6378. The study includes monitoring (at
pre-specified intervals) for clonal expansion.
Delayed Platelet Engraftment
Delayed platelet engraftment has been observed with LYFGENIA.
Bleeding risk is increased prior to platelet engraftment and may
continue after engraftment in patients with prolonged
thrombocytopenia. Two patients (4%) required more than 100 days
post treatment with LYFGENIA to achieve platelet engraftment.
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.
Neutrophil Engraftment Failure
There is a potential risk of neutrophil engraftment failure
after treatment with LYFGENIA. Neutrophil engraftment failure is
defined as failure to achieve three consecutive absolute neutrophil
counts (ANC) ≥ 0.5 × 109 cells/L obtained on different days by Day
43 after infusion of LYFGENIA. Monitor neutrophil counts until
engraftment has been achieved. If neutrophil engraftment failure
occurs in a patient treated with LYFGENIA, provide rescue treatment
with the back-up collection of CD34+ cells.
Insertional Oncogenesis
There is a potential risk of lentiviral vector-mediated
insertional oncogenesis after treatment with LYFGENIA.
Hypersensitivity Reactions
Allergic reactions may occur with the infusion of LYFGENIA. The
dimethyl sulfoxide (DMSO) or dextran 40 in LYFGENIA may cause
hypersensitivity reactions, including anaphylaxis.
Anti-retroviral Use
Patients should not take prophylactic HIV anti-retroviral
medications for at least one month prior to mobilization and until
all cycles of apheresis are completed. There are some long-acting
anti-retroviral medications that may require a longer duration of
discontinuation for elimination of the medication. If a patient is
taking anti-retrovirals for HIV prophylaxis, confirm a negative
test for HIV before beginning mobilization and apheresis of CD34+
cells.
Hydroxyurea Use
Patients should not take hydroxyurea for at least 2 months prior
to mobilization and until all cycles of apheresis are completed. If
hydroxyurea is administered between mobilization and conditioning,
discontinue 2 days prior to initiation of conditioning.
Iron Chelation
Drug-drug interactions between iron chelators and the
mobilization process and myeloablative conditioning agent must be
considered. Iron chelators should be discontinued at least 7 days
prior to initiation of mobilization or conditioning. Do not
administer myelosuppressive iron chelators (e.g., deferiprone) for
6 months post-treatment with LYFGENIA. Non-myelosuppressive iron
chelation should be restarted no sooner than 3 months after
LYFGENIA infusion. Phlebotomy can be used in lieu of iron
chelation, when appropriate.
Interference with PCR-based Testing
Patients who have received LYFGENIA are likely to test positive
by polymerase chain reaction (PCR) assays for HIV due to integrated
BB305 LVV proviral DNA, resulting in a possible false-positive PCR
assay test result for HIV. Therefore, patients who have received
LYFGENIA should not be screened for HIV infection using a PCR-based
assay.
Adverse Reactions
The most common adverse reactions ≥ Grade 3 (incidence ≥ 20%)
were stomatitis, thrombocytopenia, neutropenia, febrile
neutropenia, anemia, and leukopenia.
Three patients died during LYFGENIA clinical trials; one from
sudden cardiac death due to underlying disease and two from acute
myeloid leukemia who were treated with an earlier version of
LYFGENIA using a different manufacturing process and transplant
procedure (Study 1, Group A).
Pregnancy/Lactation
Advise patients of the risks associated with myeloablative
conditioning agents, including on pregnancy and fertility.
LYFGENIA should not be administered to women who are pregnant,
and pregnancy after LYFGENIA infusion should be discussed with the
treating physician.
LYFGENIA is not recommended for women who are breastfeeding, and
breastfeeding after LYFGENIA 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 LYFGENIA 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 LYFGENIA.
Advise patients of the options for fertility preservation.
Please see full Prescribing Information for LYFGENIA
including Boxed WARNING and Medication Guide.
About bluebird bio, Inc. bluebird bio is pursuing
curative gene therapies to give patients and their families more
bluebird days.
Founded in 2010, bluebird has been setting the standard for gene
therapy for more than a decade—first as a scientific pioneer and
now as a commercial leader. bluebird has an unrivaled track record
in bringing the promise of gene therapy out of clinical studies and
into the real-world setting, having secured FDA approvals for three
therapies in under two years. Today, we are proving and scaling the
commercial model for gene therapy and delivering innovative
solutions for access to patients, providers, and payers.
With a dedicated focus on severe genetic diseases, bluebird has
the largest and deepest ex-vivo gene therapy data set in the field,
with industry-leading programs for sickle cell disease,
β-thalassemia and cerebral adrenoleukodystrophy. 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.
bluebird continues to forge new paths as a standalone commercial
gene therapy company, 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.
LYFGENIA and bluebird bio are trademarks of bluebird bio,
Inc.
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
therapeutic potential of bluebird’s therapies, the potential for
its therapies to have a durable impact on patients, including the
potentially curative, sustained lifetime impact LYFGENIA may
provide, and the commercialization of LYFGENIA, including without
limitation, bluebird’s ability to establish commercial
infrastructure to support timely, equitable access to LYFGENIA, its
ability to successfully partner with payers, including by executing
binding agreements with payers, its expectations on timing for
activating QTCs, its expectations on the timing and size of its QTC
network and the timing of LYFGENIA’s availability at its QTCs, 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 bluebird’s 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 bluebird’s control and could cause bluebird’s 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 for the year ended December 31, 2022, as
updated by its 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: delays and challenges in bluebird’s
commercialization and manufacturing of its products; the internal
and external costs required for bluebird’s ongoing and planned
activities, and the resulting impact on expense and use of cash,
has been, and may in the future be, higher than expected which has
caused bluebird, and may in the future cause bluebird to use cash
more quickly than it expects or change or curtail some of its plans
or both; substantial doubt exists regarding bluebird’s ability to
continue as a going concern; bluebird’s expectations as to
expenses, cash usage and cash needs may prove not to be correct for
other reasons such as changes in plans or actual events being
different than bluebird’s assumptions; the risk that the efficacy
and safety results from bluebird’s prior and ongoing clinical
trials will not continue or be seen in the commercial context; the
risk that bluebird is not able to activate QTCs on the timeframe
that it expects; the risk that the QTCs experience delays in their
ability to enroll or treat patients; the risk that bluebird
experiences delays in establishing operational readiness across its
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 LYFGENIA; the risk of insertional oncogenic or
other safety events associated with lentiviral vector, drug
product, or myeloablation; and the risk that LYFGENIA will not be
successfully 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/20231208301760/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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