bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S.
Food and Drug Administration (FDA) has communicated that an
advisory committee meeting will not be scheduled for
lovotibeglogene autotemcel (lovo-cel). Lovo-cel is a potentially
transformative one-time gene therapy for individuals living with
sickle cell disease (SCD) with a proposed indication for patients
ages 12 and older who have a history of vaso-occlusive events
(VOEs). The Agency previously accepted the lovo-cel Biologics
Licensing Application (BLA) for Priority Review and set a
Prescription Drug User Fee Act (PDUFA) goal date of December 20,
2023.
“Lovo-cel is the most deeply studied gene therapy in development
for sickle cell disease and represents the third lentiviral vector
gene therapy that the Agency has reviewed from bluebird—giving us
great confidence in the robustness and maturity of our BLA
package,” said Andrew Obenshain, chief executive officer, bluebird
bio. “We remain focused on working with the Agency on its review in
anticipation of a decision by the end of this year.”
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.
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 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 individuals
living with sickle cell disease; the robustness and maturity of the
lovo-cel BLA package; the possible approval of lovo-cel by FDA and
the expected timing relating to such regulatory approval; 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; we may
encounter additional delays in the development of our programs,
including the imposition of new clinical holds, which 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 of
insertional oncogenic or other reportable events associated with
lentiviral vector, drug product, or myeloablation; the risk that
any one or more of our products or product candidates, including
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/20230816191251/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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