- European Medicines Agency Will Evaluate
LentiGlobin Marketing Authorization Application Under Accelerated
Assessment -
bluebird bio, Inc. (Nasdaq: BLUE) announced today that the
European Medicines Agency (EMA) accepted the company’s marketing
authorization application (MAA) for its investigational
LentiGlobin™ gene therapy for the treatment of adolescents and
adults with transfusion-dependent β-thalassemia (TDT) and a
non-β0/β0 genotype.
LentiGlobin was previously granted an accelerated assessment by
the Committee for Medicinal Products for Human Use (CHMP) of the
EMA in July 2018, potentially reducing the EMA’s active review time
of the MAA from 210 days to 150 days.
“People living with transfusion-dependent β-thalassemia require
frequent blood transfusions that are life-saving but may lead to
complications, including organ failure due to iron overload,” said
David Davidson, M.D., chief medical officer, bluebird bio. “The
acceptance of our marketing authorization application for
LentiGlobin is a milestone that advances us toward our goal of
providing to patients the first one-time gene therapy that
addresses the underlying genetic cause of TDT. We share this
important milestone with the patients, families and healthcare
providers who made it possible through their participation in our
pioneering clinical studies of LentiGlobin.”
The MAA for LentiGlobin is supported by data from the completed
Phase 1/2 Northstar (HGB-204) study and the ongoing Phase 1/2
HGB-205 study as well as available data from the Phase 3
Northstar-2 (HGB-207) study and the long-term follow-up study
LTF-303.
About Transfusion-Dependent β-ThalassemiaTDT is an
inherited blood disorder caused by a mutation in the β-globin gene,
which causes ineffective red blood cell production leading to
severe anemia. Supportive care for people with TDT consists of a
lifelong regimen of chronic blood transfusions to enable survival
and suppress symptoms of the disease, and iron chelation therapy to
manage iron overload that results from the transfusions.
Despite the availability of supportive care, many people with
TDT experience serious complications and organ damage due to
underlying disease and iron overload. By eliminating or reducing
the need for blood transfusions, the long-term complications
associated with TDT may be reduced.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT)
has been successfully used to treat TDT and is currently the only
available option with the potential to correct the genetic
deficiency in TDT. Complications of allo-HSCT include a risk of
treatment-related mortality, graft failure, graft-versus-host
disease (GvHD) and opportunistic infections, particularly in
patients who undergo non-sibling matched allo-HSCT.
About LentiGlobinLentiGlobin is a one-time gene therapy
being studied as a potential treatment to address the underlying
genetic cause of TDT, which could eliminate or reduce the need for
blood transfusions.
bluebird bio’s clinical development program for LentiGlobin
includes ongoing studies around the world with sites
in Australia, Germany, Greece, France, Italy, Thailand,
the United Kingdom and the United States. For more
information visit: www.northstarclinicalstudies.com or
clinicaltrials.gov using identifier NCT01745120.
In addition, bluebird is conducting a long-term safety and
efficacy follow-up study (LTF-303) for people who have participated
in bluebird bio-sponsored clinical studies of LentiGlobin for TDT
and sickle cell disease.
The EMA previously granted Priority Medicines (PRIME)
eligibility and Orphan Medicinal Product designation to LentiGlobin
for the treatment of TDT. LentiGlobin is also part of the EMA’s
Adaptive Pathways pilot program, which is part of the EMA’s effort
to improve timely access for patients to new medicines.
The U.S. Food and Drug Administration (FDA) also granted
LentiGlobin Orphan Drug status and Breakthrough Therapy designation
for the treatment of TDT.
About bluebird bio, Inc.With its lentiviral-based gene
therapies, T cell immunotherapy expertise and gene editing
capabilities, bluebird bio has built a pipeline with broad
potential application in severe genetic diseases and cancer.
bluebird bio's gene therapy clinical programs include
investigational treatments for cerebral adrenoleukodystrophy,
transfusion-dependent β-thalassemia, also known as β-thalassemia
major, and severe sickle cell disease.
bluebird bio's oncology pipeline is built upon the company's
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. The
company’s lead oncology programs are anti-BCMA CAR T programs
partnered with Celgene.
bluebird bio’s discovery research programs include utilizing
megaTAL/homing endonuclease gene editing technologies with the
potential for use across the company's pipeline.
bluebird bio has operations in Cambridge, Massachusetts;
Seattle, Washington; Durham, North Carolina and Zug,
Switzerland.
LentiGlobin is a trademark of bluebird bio, Inc.
Forward-Looking StatementsThis release contains
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995, including statements
regarding the Company’s views with respect to the potential for its
LentiGlobin product candidate to treat transfusion-dependent
ß-thalassemia, and the Company’s expectations regarding the review,
potential regulatory approval and potential commercial launch of
its LentiGlobin product candidate in the United States and Europe.
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, the risks that the preliminary positive
efficacy and safety results from our prior and ongoing clinical
trials of LentiGlobin will not continue or be repeated in our
ongoing or planned clinical trials of LentiGlobin, the risks that
the changes we have made in the LentiGlobin manufacturing will not
result in improved patient outcomes, risks that the current or
planned clinical trials of LentiGlobin will be insufficient to
support future regulatory submissions or to support marketing
approval in the US and EU, and the risk that any one or more of our
product candidates, will not be successfully developed, approved or
commercialized. 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 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20181005005057/en/
bluebird bioInvestors:Elizabeth Pingpank,
617-914-8736epingpank@bluebirdbio.comorMedia:Catherine Falcetti,
339-499-9436cfalcetti@bluebirdbio.com
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