BLA submission based on data from the largest
and most mature clinical development program for any gene therapy
in sickle cell disease
PDUFA date set for December 20, 2023
bluebird bio, Inc. (Nasdaq: BLUE) today announced that
the U.S. Food and Drug Administration (FDA) has accepted the
Biologics License Application (BLA) for lovotibeglogene autotemcel
(lovo-cel) for priority review. Lovo-cel is a potentially
transformative one-time gene therapy for individuals living with
sickle cell disease (SCD) ages 12 and older who have a history of
vaso-occlusive events (VOEs). It is specifically designed to treat
the underlying cause of SCD through the addition of a functional
gene that enables production of anti-sickling adult hemoglobin and
is the most deeply studied gene therapy in development for this
disease. The agency has set a Prescription Drug User Fee Act
(PDUFA) goal date of December 20, 2023.
“The burden that people living with SCD and their families live
with today is staggering. Beyond extreme pain crises that send
patients to the hospital, SCD progression is associated with grave
long-term consequences,” said Andrew Obenshain, chief executive
officer, bluebird bio. “The FDA’s acceptance of our BLA for
lovo-cel moves us one step closer in bringing a potentially
transformative therapy to the sickle cell disease community that is
long overdue, and we are grateful to the patients, caregivers,
researchers, clinicians, and community leaders who have enabled
this exciting milestone. We look forward to working with the agency
on its review.”
The BLA for lovo-cel is based on efficacy results from 36
patients in the HGB-206 study Group C cohort with a median 32
months of follow-up and two patients in the HGB-210 study with 18
months of follow-up each. The BLA submission also includes safety
data from 50 patients treated across the entire lovo-cel program,
including six patients with six or more years of follow-up, which
is the longest follow-up of any gene therapy program for SCD.
If approved, lovo-cel will be bluebird bio’s third ex-vivo gene
therapy approved by the FDA for a rare genetic disease and its
second FDA approval for an inherited hemoglobin disorder, building
on more than a decade of leadership in gene therapy.
The FDA’s Priority Review designation is granted to therapies
that have the potential to provide significant improvements in the
treatment, diagnosis, or prevention of serious conditions, and
targets a review timeline of six months from the time of filing,
compared to a standard review timeline of 10 months.
The FDA previously granted lovo-cel orphan drug designation,
fast track designation, regenerative medicine advanced therapy
(RMAT) designation, and rare pediatric disease designation.
About sickle cell disease (SCD)
Sickle cell disease (SCD) is a complex and progressive genetic
disease associated with debilitating and unpredictable pain crises,
anemia, irreversible damage to vital organs, and early death. In
SCD, high concentrations of sickle hemoglobin (HbS) in red blood
cells (RBCs) cause RBCs to become sickled, sticky, and rigid with a
shorter life span, which manifests acutely as hemolytic anemia,
vasculopathy, and vaso-occlusion. Pain onset can be sudden and
unpredictable, often requiring hospitalization. Fifty to sixty
percent of adults with sickle cell disease have end organ damage,
with 24 percent experiencing damage in multiple organs, and one in
four patients have a stroke by the age of 45. The impact of SCD is
pervasive and affects every aspect of life for patients and their
families and caregivers – from missed work and school, decreased
quality of life and mental health, and the ability to complete
daily tasks. In the U.S., there are approximately 100,000 people
living with SCD, and the median age of death is 45 years of
age.
While SCD was the first disease to have a genetic cause
identified, treatment advances have lagged – since that discovery
in 1949,i only four therapies have been approved,ii none of which
address the underlying genetic cause of disease.
About lovotibeglogene autotemcel (lovo-cel)
lovotibeglogene autotemcel (lovo-cel) gene therapy is an
investigational one-time treatment being studied for sickle cell
disease (SCD), that is designed to add functional copies of a
modified form of the β-globin gene (βA-T87Q-globin gene) into a
patient’s own hematopoietic (blood) stem cells (HSCs). Once
patients have the βA-T87Q-globin gene, their red blood cells (RBCs)
can produce anti-sickling hemoglobin (HbAT87Q) that decreases the
proportion of HbS, with the goal of reducing sickled RBCs,
hemolysis, and other complications. bluebird bio’s clinical
development program for lovo-cel includes the completed Phase 1/2
HGB-205 and ongoing Phase 1/2 HGB-206 and Phase 3 HGB-210 studies.
bluebird bio is also conducting a long-term safety and efficacy
follow-up study (LTF-307) for people who have been treated with
lovo-cel in bluebird bio-sponsored clinical studies.
In the BLA submission, as of August 2022, the majority of
adverse events in treated patients were attributed to underlying
sickle cell disease or conditioning with busulfan. Nonserious
adverse events related to lovo-cel included infusion reactions (hot
flush and decreased blood pressure) in two patients (2% each).
Serious adverse events related to lovo-cel included anemia in two
patients (4%) with concurrent alpha-thalassemia trait, and leukemia
in two patients (4%), not resulting from insertional oncogenesis.
Three of 50 patients (6%) died, one due to sudden cardiac death and
two due to leukemia.
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 programs for sickle cell disease, β-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 the
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.
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, including,
without limitation, our statements regarding: the therapeutic
potential of lovo-cel, including its potential as a transformative
therapy for the sickle cell disease community; the possible
approval of lovo-cel by FDA and the expected timing relating to
such regulatory approval, including the shortened FDA review period
for therapies designated for Priority Review; and bluebird bio’s
ability to pursue creative gene therapies to give patients and
their families more bluebird days. Such forward-looking statements
are based on historical performance and current expectations and
projections about our future financial results, 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 financial results, 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 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 include, but are not limited to: delays and challenges in
obtaining regulatory approval of our product candidates and our
commercialization and manufacturing of our products, including
risks associated with demonstrating analytical comparability with
respect to our lovo-cel program; we may encounter additional delays
in the development of our programs, including the imposition of new
clinical holds, that may impact our ability to meet our expected
timelines and increase our costs; the internal and external costs
required for our 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 us, and may in the
future cause us to use cash more quickly than we expect or change
or curtail some of our plans or both; our 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 from our assumptions; the risk that the efficacy and
safety results from our prior and ongoing clinical trials will not
continue or be seen in additional patients treated with our product
candidates; the risk that additional insertional oncogenic or other
reportable events associated with lentiviral vector, drug product,
or myeloablation will be discovered or reported over time; the risk
that our eli-cel, beti-cel and lovo-cel programs may be subject to
further delays in their development, including but not limited to
the imposition of new clinical holds; the risk that any one or more
of our products or product candidates, including eli-cel and,
beti-cel or lovo-cel, will not be successfully developed, approved
or commercialized, as applicable. 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.
_________________________ i Pauling L, Itano HA, Singer SJ,
Wells IC. Sickle cell anemia, a molecular disease. Science.
1949;110(2865):543-548. doi:10.1126/science.110.2865.543. ii Rai P,
Ataga KI. Drug Therapies for the Management of Sickle Cell Disease.
F1000Res. 2020 Jun 11;9:F1000 Faculty Rev-592. doi:
10.12688/f1000research.22433.1. PMID: 32765834; PMCID:
PMC7388199.
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version on businesswire.com: https://www.businesswire.com/news/home/20230621695509/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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