Severe vaso-occlusive events were eliminated
for 94% (30/32) of evaluable patients and all VOEs were eliminated
for 88% (28/32) of evaluable patients between 6 and 18 months
post-infusion
LYFGENIA is the most deeply studied gene
therapy for sickle cell disease with the most patients treated and
longest follow-up
Management to host conference call today,
December 8, 2023, at 4:15 p.m. ET
bluebird bio, Inc. (Nasdaq: BLUE) (“bluebird bio” or “bluebird”)
today announced the U.S. Food and Drug Administration (FDA) has
approved LYFGENIA™ (pronounced as ‘lif-JEN-ee-uh’) (lovotibeglogene
autotemcel), also known as lovo-cel, for the treatment of sickle
cell disease in patients ages 12 and older who have a history of
vaso-occlusive events (VOEs). LYFGENIA is a one-time gene therapy
that has the potential to resolve vaso-occlusive events and is
custom-designed to treat the underlying cause of sickle cell
disease.
"Bringing LYFGENIA to people living with sickle cell disease is
a milestone that bluebird has been working toward for almost a
decade—and one that members of the sickle cell disease community
have been waiting on for much longer,” said Andrew Obenshain, chief
executive officer, bluebird bio. “LYFGENIA has the potential to
have a transformational impact for patients who currently live
under the shadow of unpredictable and debilitating vaso-occlusive
events. This approval also marks bluebird’s third ex vivo gene
therapy approved by the FDA for a rare genetic disease and second
FDA approval for an inherited hemoglobin disorder, cementing our
position as a gene therapy leader.”
“We’re enthusiastic at the Sickle Cell Disease Association of
America Inc. about the FDA’s approval of this therapy, which could
change the lives of people and families affected by sickle cell
disease,” said Regina Hartfield, president and CEO of the Sickle
Cell Disease Association of America Inc. “As the national advocacy
organization for people with sickle cell, we’re strongly supportive
of the new potentially curative option for treatment and excited
for the future.”
“People living with sickle cell disease face potentially
devastating health consequences, diminished quality of life, and
harmful stigma as a result of their disease,” said Julie Kanter,
M.D., a LYFGENIA investigator and director of the University of
Alabama Birmingham Adult Sickle Cell Clinic and associate professor
in the Division of Hematology and Oncology. “Today we can celebrate
the availability of a potentially transformative new therapeutic
option made possible by the incredible courage of patients and
families who participated in clinical studies.”
“Sickle cell disease was the first genetic disorder to be
characterized at the molecular level a half-century ago and today –
after decades of waiting – we finally have a therapy that addresses
the underlying cause of this devastating disease,” said John
Tisdale, MD, Chief, Cellular and Molecular Therapeutics Branch at
the National Heart, Lung, and Blood Institute (NHLBI), who served
as principal investigator on the HGB-206 study and was a key
collaborator throughout the clinical development program. “The
development of this therapy has been marked by transparency and
collaboration that laid the groundwork for other technologies to
follow, and the NIH is extremely pleased to have supported this
advance.” NHLBI is part of the National Institutes of Health.
Sickle cell disease is a complex and progressive genetic disease
associated with unpredictable and debilitating vaso-occlusive
events (VOEs).1,2,3,4 In sickle cell disease, high concentrations
of sickle hemoglobin (HbS) in red blood cells (RBCs) cause them to
become misshapen, sticky and rigid, with a shorter life span, which
manifests acutely as hemolytic anemia, vasculopathy and
vaso-occlusion. The burden associated with VOEs is pervasive and
can affect every aspect of life for patients and their families and
caregivers.
LYFGENIA works by permanently 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 expected 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 vaso-occlusive
events (VOEs).
LYFGENIA will be available at bluebird bio’s established network
of Qualified Treatment Centers (QTCs), which receive specialized
training to administer complex gene therapies like LYFGENIA.
Information on bluebird’s QTC network, as well as personalized
support focused on the needs of each patient throughout their
treatment journey, is available through bluebird’s patient support
program, my bluebird support. Patients can call 833-888-NEST
(833-888-6378) or visit mybluebirdsupport.com for more
information. bluebird plans to make the therapy available in early
2024.
LYFGENIA was granted Priority Review in June 2023. The Company
did not receive a Rare Pediatric Disease Priority Review Voucher as
part of the review. LYFGENIA was previously granted orphan drug
designation, fast track designation, regenerative medicine advanced
therapy (RMAT) designation, and rare pediatric disease
designation.
Clinical Data Supporting Approval of LYFGENIA 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).
Conference Call Details bluebird bio will hold a
conference call for analysts and investors today December 8, at
4:15 p.m. ET.
To access the call via telephone, please register at this link:
https://register.vevent.com/register/BI50feb64355294f899507004a73e5ffb8
to receive a dial in number and unique PIN to access the live
conference call.
The live webcast of the call can be accessed by visiting the
“Events & Presentations” page within the Investors & Media
section of the bluebird website at http://investor.bluebirdbio.com.
A replay of the webcast will be available on the bluebird website
for 90 days following the event.
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 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 LYFGENIA, including the transformative and
potentially curative impact it may have for patients; the
commercialization of LYFGENIA, including without limitation, its
availability, and the timing thereof, at 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 its
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 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.
______________________________ 1 Kato, et al. Nat Rev Dis
Primers. 2018;4:(18010):1-22. 2 Powars DR, et al. Medicine.
2005;84(6):363–376. 3 Chaturvedi S, et al. Am J Hematol.
2018;93:1153–1160. 4 Elmariah H, et al. Am J Hematol.
2014;89(5):530-535.
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version on businesswire.com: https://www.businesswire.com/news/home/20231208492260/en/
Investors & Media: Investors: Courtney O’Leary,
978-621-7347 coleary@bluebirdbio.com
Media: Jess Rowlands, 857-299-6103
jess.rowlands@bluebirdbio.com
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