EMA’s Pharmacovigilance Risk Assessment
Committee (PRAC) confirms favorable benefit-risk balance of
ZYNTEGLO
Company has informed EMA of lift of voluntary
temporary marketing suspension
bluebird bio, Inc. (Nasdaq: BLUE) today announced that
the European Medicines Agency's (EMA) Pharmacovigilance Risk
Assessment Committee (PRAC) has concluded based on the review of
all available data that the benefit-risk balance of medicinal
products containing ZYNTEGLO™ (betibeglogene autotemcel gene
therapy) remains favorable. As of today, bluebird bio has informed
the EMA that the company is lifting the voluntary marketing
suspension.
“Patient safety remains our top priority. To this end, we are
grateful to the PRAC for its comprehensive review of the available
evidence and positive recommendation for ZYNTEGLO,” said Andrew
Obenshain, president, severe genetic diseases, bluebird bio. “We
are pleased to resume offering ZYNTEGLO to patients living with
transfusion-dependent β-thalassemia, offering the potential to live
free from transfusions which is evidenced by our clinical studies
where patients are maintaining normal or near-normal hemoglobin
levels over the course of up to seven years of follow-up.”
No cases of hematologic malignancy have been reported in any
patient who has received treatment with ZYNTEGLO. However because
it is manufactured using the same BB305 lentiviral vector used in
LentiGlobin for sickle cell disease (SCD; investigational drug
product bb1111), bluebird bio decided to temporarily suspend
marketing of ZYNTEGLO while the root cause of the safety events
reported earlier this year for LentiGlobin for SCD were
investigated by the company and assessed by the PRAC.
As previously announced on June 7, 2021, the U.S. Food and Drug
Administration (FDA) lifted the clinical holds on the Phase 1/2
HGB-206 and Phase 3 HGB-210 studies of LentiGlobin for SCD
following the Agency’s review of the data.
The recommendation from the PRAC can be viewed here on the EMA
website. As a next step, the recommendation will be forwarded to
the Committee for Advanced Therapies (CAT) and Committee for
Medicinal Products for Human Use (CHMP) for adoption. The final
stage of the review procedure is the adoption by the European
Commission (EC) of a legally binding decision applicable in all EU
Member States.
About ZYNTEGLO (betibeglogene autotemcel; beti-cel)
Betibeglogene autotemcel (beti-cel) is a one-time gene therapy
that adds 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 a patient has the βA-T87Q-globin gene, they
have the potential to produce HbAT87Q, which is gene
therapy-derived adult hemoglobin (Hb), at levels that may eliminate
or significantly reduce the need for transfusions. In studies of
beti-cel, transfusion independence (TI) is defined as no longer
needing red blood cell transfusions for at least 12 months while
maintaining a weighted average Hb of at least 9 g/dL.
beti-cel is manufactured using the BB305 lentiviral vector
(LVV), a third-generation, self-inactivating LVV. The promoter, a
regulatory element of the LVV that controls the expression of the
transgene, selected for BB305 is a cellular (non-viral) promoter
that drives gene expression only in the erythroid lineage cells
(red blood cells and their precursors).
The European Commission granted conditional marketing
authorization (CMA) for beti-cel, marketed as ZYNTEGLO™ gene
therapy, for patients 12 years and older with TDT who do not have a
β0/β0 genotype, for whom hematopoietic stem cell (HSC)
transplantation is appropriate, but a human leukocyte antigen
(HLA)-matched related HSC donor is not available. Non-serious
adverse events (AEs) observed during clinical studies that were
attributed to beti-cel included abdominal pain, thrombocytopenia,
leukopenia, neutropenia, hot flush, dyspnea, pain in extremity,
tachycardia and non-cardiac chest pain. One serious adverse event
(SAE) of thrombocytopenia was considered possibly related to
beti-cel.
Additional AEs observed in clinical studies were consistent with
the known side effects of HSC collection and bone marrow ablation
with busulfan, including SAEs of veno-occlusive disease. For
details, please see the product information, containing Summary
of Product Characteristics (SmPC).
On April 28, 2020, the EMA renewed the CMA for beti-cel. The CMA
for beti-cel is valid in the 27 member states of the EU as well as
Iceland, Liechtenstein and Norway. In November 2020, bluebird bio
submitted to the EMA an application for the second renewal of the
CMA. ZYNTEGLO also has a CMA in Great Britain, further to the
grandfathering of the license by the MHRA earlier this year.
The U.S. Food and Drug Administration (FDA) granted beti-cel
Orphan Drug status and Breakthrough Therapy designation for the
treatment of TDT.
bluebird bio is on track to complete its rolling Biologics
License Application (BLA) submission to the FDA for beti-cel in
mid-2021. This submission is anticipated to include adult,
adolescent and children with transfusion dependent β-thalassemia
across all genotypes (including non-β0/β0 genotypes and β0/β0
genotypes). Beti-cel is not approved in the U.S.
Beti-cel continues to be evaluated in the ongoing Phase 3
Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies. bluebird
bio 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 beti-cel.
About bluebird bio, Inc.
bluebird bio, Inc. (NASDAQ: BLUE) is pioneering gene therapy
with purpose. From our Cambridge, Mass., headquarters, we’re
developing gene and cell therapies for severe genetic diseases and
cancer, with the goal that people facing potentially fatal
conditions with limited treatment options can live their lives
fully. Beyond our labs, we’re working to positively disrupt the
healthcare system to create access, transparency and education so
that gene therapy can become available to all those who can
benefit.
bluebird bio is a human company powered by human stories. We’re
putting our care and expertise to work across a spectrum of
disorders including cerebral adrenoleukodystrophy, sickle cell
disease, β-thalassemia and multiple myeloma using three gene
therapy technologies: gene addition, cell therapy and
(megaTAL-enabled) gene editing.
bluebird bio has additional nests in Seattle, Wash.; Durham,
N.C.; and Zug, Switzerland. For more information, visit
bluebirdbio.com.
Follow bluebird bio on social media: @bluebirdbio,
LinkedIn, Instagram and YouTube.
ZYNTEGLO and bluebird bio are trademarks 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, including
statements regarding the company’s expectations regarding the
commercialization of ZYNTEGLO, and the timing of the submission of
a BLA for beti-cel to the FDA. 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, which include but are not limited to: the risk that
insertional oncogenic events associated with lentiviral vector or
additional MDS events associated with transplant or myeloablation
will be discovered or reported over time; the risk that insertional
oncogenic events associated with lentiviral vector in other
programs may result in a clinical hold of our programs in SCD, TDT
or cerebral adrenoleukodystrophy; the risk that we may not be able
to execute on our business plans, including meeting our expected or
planned regulatory milestones, submissions or timelines, such as in
the completion of our BLA submission for beti-cel and the renewal
of the CMA for beti-cel; the risk that LentiGlobin for SCD or
beti-cel will not be approved for marketing by the FDA, and the
risk that we will not successfully bring LentiGlobin for SCD or
beti-cel to market in the United States; the risk that we may not
resume patient treatment with ZYNTEGLO in the commercial context in
a timely manner or at all; the risk that results seen in our
clinical trials of beti-cel may not be seen in the commercial
context for ZYNTEGLO; and the risk that with the impact on the
execution and timing of our business plans, we may not successfully
execute our previously-announced plans to spin-off our oncology
portfolio and programs into an independent publicly-traded company
on the timeline that we expect, or at all. 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 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. 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/20210709005202/en/
Investors: Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com
or
Media: Jenn Snyder, 617-448-0281
jsnyder@bluebirdbio.com
Victoria von Rinteln, 617-914-8774
vvonrinteln@bluebirdbio.com
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