First agreements with statutory health
insurances utilize bluebird’s innovative value-based payment model
and provide coverage for ZYNTEGLO for up to 50% of patients in
Germany
First qualified treatment center established at
University Hospital of Heidelberg to provide ZYNTEGLO to
patients
bluebird bio, Inc. (Nasdaq: BLUE) announced the launch in
Germany of 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. This is the first
time ZYNTEGLO is commercially available.
TDT is a severe genetic disease caused by mutations in the
β-globin gene that result in significantly reduced or absent adult
hemoglobin (HbA). In order to survive, people with TDT maintain
hemoglobin (Hb) levels through lifelong chronic blood transfusions.
These transfusions carry the risk of progressive multi-organ damage
due to unavoidable iron overload. ZYNTEGLO is a one-time gene
therapy that addresses the underlying genetic cause of TDT and
offers patients the potential to become transfusion independent,
which, once achieved, is expected to be lifelong.
Due to the highly technical and specialized nature of
administering gene therapy in rare diseases, bluebird bio is
working with institutions that have expertise in stem cell
transplant as well as in treating patients with TDT to create
qualified treatment centers that will administer ZYNTEGLO. bluebird
bio has established a collaboration with University Hospital of
Heidelberg as the first qualified treatment center in Germany.
In addition, bluebird has entered into value-based payment
agreements with multiple statutory health insurances in Germany to
help ensure patients and their healthcare providers have access to
ZYNTEGLO and that payers only pay if the therapy delivers on its
promise. bluebird’s proposed innovative model is limited to five
payments made in equal installments. An initial payment is made at
the time of infusion. The four additional annual payments are only
made if no transfusions for TDT are required for the patient.
“For patients with TDT, lifelong chronic blood transfusions are
required in order to survive. We are thrilled to announce that
ZYNTEGLO will now be available for patients in the EU living with
this severe disease,” says Alison Finger, chief commercial officer,
bluebird bio. “In addition to confirming manufacturing readiness of
our partner, apceth Biopharma GmbH, bluebird has also submitted a
dossier to the Joint Federal Committee (G-BA) in Germany for drug
benefit assessment. We would like to thank our collaborators for
their commitment in helping us transform the healthcare system by
accepting innovative payment models, and we look forward to
treating our first commercial patient soon.”
About LentiGlobin for β-Thalassemia (autologous CD34+ cells
encoding βA-T87Q-globin gene)
The European Commission granted conditional marketing
authorization for LentiGlobin for β-thalassemia, to be marketed as
ZYNTEGLO™ (autologous CD34+ cells encoding βA-T87Q-globin gene)
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.
TDT is a severe genetic disease caused by mutations in the
β-globin gene that result in reduced or significantly reduced
hemoglobin (Hb). In order to survive, people with TDT maintain Hb
levels through lifelong chronic blood transfusions. These
transfusions carry the risk of progressive multi-organ damage due
to unavoidable iron overload.
LentiGlobin for β-thalassemia 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 hemoglobin, at
levels that may eliminate or significantly reduce the need for
transfusions.
Non-serious adverse events (AEs) observed during the HGB-204,
HGB-207 and HGB-212 clinical studies that were attributed to
LentiGlobin for β-thalassemia were hot flush, dyspnoea, abdominal
pain, pain in extremities, thrombocytopenia, leukopenia,
neutropenia and non-cardiac chest pain. One serious adverse event
(SAE) of thrombocytopenia was considered possibly related to
LentiGlobin for β-thalassemia for TDT.
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.
The conditional marketing authorization for ZYNTEGLO is valid in
the 28 member states of the EU as well as Iceland, Liechtenstein
and Norway. For details, please see the Summary of Product
Characteristics (SmPC).
The U.S. Food and Drug Administration (FDA) granted LentiGlobin
for β-thalassemia Orphan Drug status and Breakthrough Therapy
designation for the treatment of TDT. LentiGlobin for β-thalassemia
is not approved in the United States.
bluebird bio has initiated the rolling BLA submission for
approval in the U.S., and is engaged with the FDA in discussions
regarding the requirements and timing of the various components of
the rolling BLA submission. Subject to these ongoing discussions,
the company is currently planning to complete the BLA submission in
the first half of 2020.
LentiGlobin for β-thalassemia continues to be evaluated in the
ongoing Phase 3 Northstar-2 and Northstar-3 studies. For more
information about the ongoing clinical studies, visit
www.northstarclinicalstudies.com or clinicaltrials.gov and use
identifier NCT02906202 for Northstar-2 (HGB-207) or NCT03207009 for
Northstar-3 (HGB-212).
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 LentiGlobin for
β-thalassemia. For more information visit:
https://www.bluebirdbio.com/our-science/clinical-trials or
clinicaltrials.gov and use identifier NCT02633943 for 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 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, LentiGlobin, and bluebird bio 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 regarding the Company’s plans and expectations
for the commercialization for ZYNTEGLO™ (autologous CD34+ cells
encoding βA-T87Q-globin gene, formerly LentiGlobin™ in TDT) to
treat TDT, and the potential implications of clinical data for
patients. 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 the efficacy and
safety results from our prior and ongoing clinical trials of
ZYNTEGLO will not continue or be repeated in our ongoing or planned
clinical trials of ZYNTEGLO; the risk that the current or planned
clinical trials of ZYNTEGLO will be insufficient to support
regulatory submissions or marketing approval in the US, or for
additional patient populations in the EU; the risk that the
production of HbAT87Q may not be sustained over extended periods of
time; the risk that we may not secure adequate pricing or
reimbursement to support continued development or commercialization
of ZYNTEGLO; the risk that our collaborations with qualified
treatment centers will not continue or be successful; and that the
risk that commercial patients treated with ZYNTEGLO will not
achieve or maintain transfusion independence. 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200113005374/en/
bluebird bio Investors: Elizabeth Pingpank, 617-914-8736
epingpank@bluebirdbio.com or Media: Jennifer Snyder,
617-448-0281 jsnyder@bluebirdbio.com
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