European Medicines Agency Approves Refined Commercial Manufacturing Specifications for ZYNTEGLO™
October 22 2019 - 07:30AM
Business Wire
First gene therapy for patients 12 years and
older with transfusion-dependent β-thalassemia who do not have a
β0/β0 genotype now available to be manufactured in the European
Union
bluebird bio, Inc. (Nasdaq: BLUE) announced today that the
European Medicines Agency (EMA) approved the refined commercial
drug product manufacturing specifications for ZYNTEGLO™ (autologous
CD34+ cells encoding βA-T87Q-globin gene), a one-time gene therapy
for patients 12 years and older with transfusion-dependent
β-thalassemia (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.
“We look forward to serving TDT patients with ZYNTEGLO and
providing a treatment option that offers the possibility of a
transfusion-free future,” said Alison Finger, chief commercial
officer, bluebird bio. “This is one step along the commercial
journey as we advance our ongoing launch and market access
activities on a country-by-country basis, with the goal of
enrolling our first commercial patient in 2019.”
The refined commercial drug product specifications support the
efficacy and safety profile of ZYNTEGLO and will give patients the
best opportunity for clinically meaningful outcomes consistent with
the results that were foundational to the conditional marketing
authorization in the European Union. ZYNTEGLO addresses the
underlying genetic cause of TDT and offers patients the potential
to become transfusion independent, which once achieved is expected
to be life-long.
“These are exciting times also for apceth, as we are now in the
final stages of preparing to manufacture a cell-based gene therapy
for commercial use,” commented Dr. Christine Guenther, CEO of
apceth Biopharma. “We are proud to be the commercial manufacturing
partner of bluebird bio and to be part of bringing this potentially
life-changing therapy to TDT patients in Europe.”
Data Supporting Clinical Profile of ZYNTEGLO
The conditional marketing authorization is supported by
efficacy, safety and durability data from the Phase 1/2 HGB-205
study and the completed Phase 1/2 Northstar (HGB-204) study as well
as available data from the ongoing Phase 3 Northstar-2 (HGB-207)
and Northstar-3 (HGB-212) studies, and the long-term follow-up
study LTF-303, as of the data cut off of December 13, 2018.
Non-serious adverse events (AEs) observed during clinical trials
that were attributed to ZYNTEGLO were hot flush, dyspnoea,
abdominal pain, pain in extremities and non-cardiac chest pain. One
serious adverse event (SAE) of thrombocytopenia was considered
possibly related to ZYNTEGLO.
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. At last
follow up all patients treated with ZYNTEGLO in the clinical trial
program remain alive.
For details, please see the Summary of Product Characteristics
(SmPC). ZYNTEGLO has received a conditional marketing authorization
in the European Union and is not approved in the United States.
ZYNTEGLO continues to be evaluated in the ongoing Phase 3
Northstar-2 and Northstar-3 studies and the long-term follow-up
study LTF-303.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
Cambridge, Mass., headquarters, we’re developing gene 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 by researching cerebral adrenoleukodystrophy, sickle cell
disease, transfusion-dependent 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, LentiGlobin and Lenti-D are trademarks of bluebird
bio, Inc.
The full common name for ZYNTEGLO: A genetically modified
autologous CD34+ cell enriched population that contains
hematopoietic stem cells transduced with lentiviral vector encoding
the βA-T87Q-globin gene.
Forward-Looking Statements
This release contains “forward-looking statements” within the
meaning of the Private Securities Litigation Reform Act of 1995,
including statements the company’s commercialization plans and
expectations for ZYNTEGLO. 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 risk that we
will encounter challenges in the commercial launch of ZYNTEGLO in
the European Union, including in managing our complex supply chain
for the delivery of drug product, in the adoption of value-based
payment models or in obtaining sufficient coverage or reimbursement
for our product. 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/20191022005380/en/
Investors & Media Investors: bluebird bio Elizabeth
Pingpank, 617-914-8736 epingpank@bluebirdbio.com Media: bluebird
bio Jenn Snyder, 617-448-0281 jsnyder@bluebirdbio.com
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