LentiGlobin™ Investigational Gene Therapy for Transfusion-Dependent Beta-Thalassemia Accepted into European Medicines Agenc...
September 21 2016 - 8:30AM
Business Wire
bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company
committed to developing potentially transformative gene therapies
for severe genetic diseases and T cell-based immunotherapies for
cancer, today announced that the European Medicines Agency (EMA)
has granted access to its Priority Medicines (PRIME) scheme for
LentiGlobin drug product in the treatment of patients with
transfusion-dependent beta-thalassemia (TDT).
The PRIME initiative provides enhanced support and increased
interaction to companies, with the goal of optimizing development
plans and speeding regulatory evaluations to potentially bring
innovative medicines to patients more quickly. To be accepted for
PRIME, a therapy must demonstrate potential to benefit patients
with unmet medical need through early clinical data or nonclinical
data. Access to the PRIME initiative complements bluebird’s ongoing
participation in the EMA’s Adaptive Pathways Pilot program, which
also aims to expedite patient access to therapies with the
potential to treat serious conditions with unmet need. It uses the
existing EU regulatory framework for medicines, including
conditional approval.
“PRIME designation will allow bluebird bio to further improve
our communication with European regulators as we continue to refine
our evidence generation plan in the context of adaptive biomedical
innovation. Overall, we believe this will enable us to accelerate
development of LentiGlobin drug product for patients with
transfusion-dependent beta thalassemia, a life-shortening disease
with significant unmet medical need,” said David Davidson, M.D.,
chief medical officer, bluebird bio. “Earlier this year we
completed enrollment in the Northstar (HGB-204) global clinical
study of LentiGlobin drug product in patients with TDT, which along
with the supporting HGB-205 study, will form the basis of our
eventual application for conditional approval in the EU under the
Adaptive Pathways Pilot program. As the data from both studies
mature, we look forward to continuing to work with the EMA to bring
LentiGlobin to patients who may benefit from gene therapy.”
About bluebird bio, Inc.
With its lentiviral-based gene therapies, T cell immunotherapy
expertise and gene editing capabilities, bluebird bio has built an
integrated product platform with broad potential application to
severe genetic diseases and cancer. bluebird bio’s gene therapy
clinical programs include its Lenti-D™ product candidate,
currently in a Phase 2/3 study, called the Starbeam Study, for the
treatment of cerebral adrenoleukodystrophy, and its
LentiGlobin™ BB305 product candidate, currently in four
clinical studies for the treatment of transfusion-dependent
ß-thalassemia, and severe sickle cell disease. bluebird bio’s
oncology pipeline is built upon the company’s leadership in
lentiviral gene delivery and T cell engineering, with a focus on
developing novel T cell-based immunotherapies, including chimeric
antigen receptor (CAR T) and T cell receptor (TCR) therapies.
bluebird bio’s lead oncology program, bb2121, is an anti-BCMA CAR T
program partnered with Celgene. bb2121 is currently being
studied in a Phase 1 trial for the treatment of relapsed/refractory
multiple myeloma. bluebird bio also has discovery research programs
utilizing megaTALs/homing endonuclease gene editing technologies
with the potential for use across the company’s pipeline.
bluebird bio has operations in Cambridge,
Massachusetts; Seattle, Washington; and Paris,
France.
Forward-Looking StatementsThis release contains
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995, including statements
regarding Company’s EU regulatory plans for its LentiGlobin BB305
product candidate to treat transfusion-dependent ß-thalassemia,
including whether the current or planned clinical trials of
LentiGlobin will be sufficient to support regulatory submissions
for marketing approval, the expected timing of any such submissions
and decisions, and any potential for an accelerated assessment of
any future MAA for LentiGlobin. In addition, it should be noted
that the EMA Adaptive Pathways program is a pilot program, and as
such there is limited information and precedent regarding the
potential outcomes for sponsors that participate in this program.
Any forward-looking statements are based on management’s current
expectations of future events and are subject to a number of risks
and uncertainties that could cause actual results to differ
materially and adversely from those set forth in or implied by such
forward-looking statements. These risks and uncertainties include,
but are not limited to, risks that the preliminary results from our
prior clinical trials of LentiGlobin will not continue or be
repeated in our current or planned clinical trials, the risk of
cessation or delay of any of the ongoing or planned clinical trials
of LentiGlobin, the risk that the EMA will not deem the MAA for
LentiGlobin sufficient for early or conditional approval in
transfusion-dependent ß-thalassemia. For a discussion of other
risks and uncertainties, and other important factors, any of which
could cause our actual results to differ from those contained in
the forward-looking statements, see the section entitled “Risk
Factors” in our most recent quarterly report on Form 10-Q, as well
as discussions of potential risks, uncertainties, and other
important factors in our subsequent filings with the Securities and
Exchange Commission. All information in this press release is as of
the date of the release, and bluebird bio undertakes no duty to
update this information unless required by law.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20160921005213/en/
bluebird bio, Inc.Investor RelationsManisha Pai,
617-245-2107mpai@bluebirdbio.comorMedia ContactsElizabeth
Pingpank, 617-914-8736epingpank@bluebirdbio.comorPure
Communications, Inc.Dan Budwick, 973-271-6085
bluebird bio (NASDAQ:BLUE)
Historical Stock Chart
From Apr 2024 to May 2024
bluebird bio (NASDAQ:BLUE)
Historical Stock Chart
From May 2023 to May 2024