Company and CMMI agreed to key terms to be
offered to State Medicaid Agencies to support access to LYFGENIA™
gene therapy for sickle cell disease
States can opt in to an outcomes-based
agreement with bluebird through the Model until February 28,
2025
bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio”) today
announced it has reached an agreement with the Center for Medicare
and Medicaid Innovation to offer an outcomes-based agreement for
LYFGENIA gene therapy under the Cell and Gene Therapy (CGT) Access
Model. LYFGENIA is a one-time gene therapy approved for the
treatment of patients 12 years of age and older with sickle cell
disease and a history of vaso-occlusive events (VOEs).
“Ensuring timely, equitable access to gene therapy for people
living with sickle cell disease insured through Medicaid has been a
cornerstone of our commercial approach since approval, and we are
pleased to build on this commitment by offering an outcomes-based
agreement to state Medicaid agencies through the Cell and Gene
Therapy Access Model,” said Tom Klima, Chief Commercial &
Operating Officer at bluebird bio. “We are also pleased to be able
to offer fertility preservation support to eligible patients
insured by Medicaid in states that opt into the Model, as we
recognize these services address an important potential barrier to
access.”
Participation in the CGT Access Model builds on bluebird’s
established leadership in developing and implementing innovative
outcomes-based agreements and has the potential to further
strengthen the coverage landscape for LYFGENIA. To date, more than
half of all states have affirmed coverage for LYFGENIA through
Preferred Drug Lists or published coverage policies, and over 50%
of Medicaid-insured individuals with sickle cell disease in the
U.S. live in a state that has approved at least one prior
authorization for LYFGENIA.
bluebird currently offers two outcomes-based agreements for
LYFGENIA to State Medicaid Agencies, and the CGT Access Model may
be a bandwidth extender for states that are unable to implement
outcomes-based agreements on their own or in a timely fashion.
bluebird’s offering through the CGT Access Model is rooted in its
validated principles of meaningful risk sharing for payers, and
tying performance to clinically meaningful outcomes that are easily
tracked through established data sources over a defined period of
time.
The model will enroll patients for a period of 6 years and
individual patients will be followed for 5 years to track
performance-related outcomes. Participation in the CGT Access Model
is voluntary and open to all states that participate in the
Medicaid Drug Rebate Program (MDRP). With key terms in place,
states have until February 28, 2025 to opt-in to participate and
states may begin participation in the Model between January 2025
and January 2026.
About LYFGENIA™ (lovotibeglogene autotemcel) or
lovo-cel
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 and progenitor cells
(HSPCs). 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
VOEs.
The Phase 1/2 HGB-206 study of LYFGENIA is 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.
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 bluebird’s ability to ensure timely, equitable
access to gene therapy; and the potential impact of the CGT Access
Model on the coverage landscape for LYFGENIA. 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’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, 2023, 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 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; the risk that there is not sufficient patient demand
or payer reimbursement to support continued commercialization of
bluebird’s therapies; the risk of insertional oncogenic or other
safety events associated with lentiviral vector, drug product, or
myeloablation, including the risk of hematologic malignancy; and
the risk that bluebird’s therapies 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/20241203868705/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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