Current standard of care relies on regular red
blood cell transfusions and iron management that carry the risk of
progressive multi-organ damage and increased risk of morbidity and
mortality
If approved, beti-cel will be the first
potentially curative gene therapy option for people with
beta-thalassemia who require regular red blood cell transfusions
and the first ex-vivo LVV gene therapy available in the U.S.
PDUFA goal date is set for August 19, 2022
bluebird bio, Inc. (Nasdaq: BLUE) today announced the
outcome of the U.S. Food and Drug Administration’s (FDA) Cellular,
Tissue, and Gene Therapies Advisory Committee (CTGTAC) discussion
of betibeglogene autotemcel (beti-cel) for the treatment of people
with beta-thalassemia who require regular red blood cell (RBC)
transfusions.
On the question “Do the benefits of beti-cel outweigh the risks
for the treatment of subjects with transfusion-dependent
beta-thalassemia?” the CTGTAC voted 13 (yes) to 0 (no).
“Despite advances in care, people living with the most severe
form of beta-thalassemia still require frequent transfusions of
healthy red blood cells to survive, tethering them to the
healthcare system for life and increasing their risk for severe
complications and early death,” said Andrew Obenshain, chief
executive officer, bluebird bio. “Today’s advisory committee
recommendation is recognition of the substantial body of clinical
data that support beti-cel as a potentially curative treatment
option for these patients. We are grateful to the members of the
beta-thalassemia community who contributed to today’s discussion
and remain committed to working with the FDA as it completes its
review of the beti-cel Biologics License Application.”
Beta-thalassemia is a rare genetic blood disease caused by
mutations in the beta-globin gene and is characterized by
significantly reduced or absent adult hemoglobin production.
Patients with the most severe form experience severe anemia and
lifelong dependence on red blood cell transfusions, a lengthy
process that patients typically undergo every 2-5 weeks. Despite
advances in treatment and improved transfusion techniques,
transfusions only temporarily address symptoms of anemia and people
with beta-thalassemia who require regular transfusions have an
increased risk for morbidity and mortality due to treatment- and
disease-related iron overload and its complications. Data from the
Cooley’s Anemia Foundation indicate that the median age of death of
U.S. transfusion-dependent beta-thalassemia patients who died
during the last decade was just 37 years.
The advisory committee’s recommendation is based on the
Biologics License Application (BLA) currently under priority review
by the FDA with a decision goal date set for August 19, 2022. The
BLA is based on data from bluebird bio’s Phase 3 studies HGB-207
(Northstar-2) and HGB-212 (Northstar-3), the Phase 1/2 HGB-204
(Northstar) and HGB-205 studies, and the long-term follow-up study
LTF-303 as of March 2021. Additionally, as of the latest data
cutoff date (August 2021), data from bluebird bio’s clinical
development program represent 240 patient-years of experience with
beti-cel and the longest available follow-up data in
beta-thalassemia patients requiring regular RBC transfusions
treated with a one-time gene therapy.
On June 9, the CTGTAC unanimously endorsed eli-cel, an
investigational LVV gene therapy for the treatment of cerebral
adrenoleukodystrophy, in a 15 to 0 vote.
“We are pleased that the advisory committee recognized the
significant potential of LVV gene therapies to address severe unmet
medical needs – demonstrated over more than a decade of research
and more than 500 patient-years of experience – as well as the
urgent need for improved treatments,” said Anne-Virginie Eggimann,
chief regulatory officer, bluebird bio. “We thank the FDA for
convening this discussion on bluebird bio’s LVV gene therapies and
look forward to the completion of the Agency’s review.”
In addition to granting the beti-cel BLA priority review, the
FDA previously granted beti-cel Orphan Drug status and Breakthrough
Therapy designation. bluebird bio is eligible to receive a priority
review voucher upon potential approval of beti-cel.
About beta-thalassemia
Beta-thalassemia is a severe genetic blood disease caused by
mutations in the beta-globin gene and is characterized by
significantly reduced or absent adult hemoglobin production. This
can result in severe anemia and lifelong dependence on red blood
cell (RBC) transfusions. Patients who require regular RBC
transfusions to maintain adequate Hb levels typically undergo the
4-7-hour process every 2-5 weeks. While transfusions temporarily
relieve symptoms associated with severe anemia, including fatigue,
weakness, and shortness of breath, they do not address the
underlying genetic cause of beta-thalassemia and can lead to
unavoidable iron overload and serious complications, including
progressive multi-organ damage and organ failure. Iron overload
resulting from beta-thalassemia or ongoing RBC transfusions
requires chronic treatment with chelation therapy; even with
chelation therapy, some patients remain significantly iron
overloaded, and only 63% of patients are adherent, due in part to
tolerability issues. Despite advances in treatment and improved
transfusion techniques, people with beta-thalassemia who require
regular transfusions have an increased risk for morbidity and
mortality.
About betibeglogene autotemcel (beti-cel)
betibeglogene autotemcel (beti-cel) (pronounced BEH tee cell) is
a one-time gene therapy custom-designed to treat the underlying
cause of beta-thalassemia in patients who require regular red blood
cell (RBC) transfusions. beti-cel adds functional copies of a
modified form of the beta-globin gene (βA-T87Q-globin gene) into a
patient’s own hematopoietic (blood) stem cells (HSCs) in order to
correct the deficiency of adult hemoglobin that is the hallmark of
beta-thalassemia. Once a patient has the βA-T87Q-globin gene, they
have the potential to produce beti-cel-derived adult hemoglobin
(HbAT87Q) at levels that may eliminate the need for transfusions.
As of the data cutoff date (August 2021), 89% (34/38) of evaluable
patients in Phase 3 beti-cel studies achieved transfusion
independence, which is defined as no longer needing RBC
transfusions for at least 12 months while maintaining a weighted
average Hb of at least 9 g/dL. These results were observed across
all ages and genotypes, including pediatric patients as young as
four years of age and those with the most severe (β0/β0)
genotypes.
beti-cel is manufactured using the BB305 lentiviral vector
(LVV), a third-generation, self-inactivating LVV that has been
studied for more than a decade across two therapeutic areas.
Adverse reactions considered related to beti-cel were infrequent
and consisted primarily of non-serious infusion-related reactions
that occurred on the day of infusion (e.g., abdominal pain, hot
flush, dyspnea, tachycardia and non-cardiac chest pain) and
cytopenias (e.g., thrombocytopenia, leukopenia and neutropenia).
One of these adverse reactions was a serious adverse event (SAE) of
thrombocytopenia considered possibly related to beti-cel and has
resolved.
The majority of AEs and SAEs in the beti-cel clinical
development program were unrelated to beti-cel and largely reflect
the known side effects of HSC collection and busulfan conditioning
regimen (including several SAEs of veno-occlusive disease that
resolved with treatment).
The Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212)
studies evaluating beti-cel are ongoing; enrollment is complete,
and all patients have been treated. bluebird bio is also conducting
a long-term follow-up study, LTF-303, to monitor safety and
efficacy for people who have participated in bluebird bio-sponsored
beti-cel clinical studies through 15 years post-treatment.
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.
bluebird bio is a trademark 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
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; 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 any one or more
of our product candidates 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20220610005588/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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