bluebird bio Enters into Advance Agreement to Sell Priority Review Voucher, if Granted, for $103 Million
30 Ottobre 2023 - 1:00PM
Business Wire
- Non-dilutive capital would strengthen
Company’s financial position -
bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the
“Company”) today announced that it has entered into an agreement to
sell a Rare Pediatric Disease Priority Review Voucher (PRV), if
received, in connection with the potential approval of
lovotibeglogene autotemcel (lovo-cel) for sickle cell disease.
Under the terms of the agreement, rights to the PRV will transfer
to the buyer and the Company will receive $103 million upon closing
of the sale, which is contingent upon the U.S. Food and Drug
Administration’s (FDA’s) approval of the biologics license
application (BLA) for lovo-cel and granting of the PRV.
The Company’s BLA for lovo-cel was previously accepted for
priority review by the FDA for patients with sickle cell disease
ages 12 and older who have a history of vaso-occlusive events
(VOEs) and has a Prescription Drug User Fee Act (PDUFA) goal date
of December 20, 2023. bluebird may be eligible for a PRV should
lovo-cel be approved for patients under the age of 18.
“The potential sale of a priority review voucher would provide
an important source of non-dilutive capital for bluebird ahead of
the anticipated launch of lovo-cel,” said Chris Krawtschuk, chief
financial officer, bluebird bio. “As the FDA completes its review
of lovo-cel, our team remains confident in the robustness and
maturity of our BLA package for individuals 12 and older with
sickle cell disease and looks forward to a regulatory decision by
the end of this year.”
Closing of the transaction remains subject to the approval of
lovo-cel and receipt of a PRV from the FDA, as well as customary
closing conditions.
About the Rare Pediatric Disease Priority Review Voucher
Program
The Rare Pediatric Disease Priority Review Voucher Program is
intended to encourage development of new drug and biological
products for the prevention and treatment of certain rare pediatric
diseases. Under this program, upon approval, the US Food and Drug
Administration awards priority review vouchers to sponsors of rare
pediatric disease product applications that meet certain criteria.
The voucher can be redeemed to receive priority review of a
subsequent marketing application for a different product. PRVs may
be sold or transferred, and there is no limit on the number of
times a PRV can be transferred.
About bluebird bio, Inc.
bluebird bio is pursuing curative gene therapies to give
patients and their families more bluebird days.
With a dedicated focus on severe genetic diseases, bluebird has
industry-leading programs for sickle cell disease, β-thalassemia
and cerebral adrenoleukodystrophy and is advancing research to
apply new technologies to these and other diseases. We custom
design each of our therapies to address the underlying cause of
disease and have developed in-depth and effective analytical
methods to understand the safety of our lentiviral vector
technologies and drive the field of gene therapy forward.
Founded in 2010, bluebird has the largest and deepest ex-vivo
gene therapy data set in the world—setting the standard for the
industry. Today, bluebird continues to forge new paths, combining
our real-world experience with a deep commitment to patient
communities and a people-centric culture that attracts and grows a
diverse flock of dedicated birds.
bluebird bio is a trademark of bluebird bio, Inc.
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
our statements regarding the closing of the sale of the Company’s
Priority Review Voucher, if received, and the Company’s financial
condition, as well as statements regarding the expected timing
relating to its potential regulatory approval and the commercial
launch of lovo-cel, if approved, the robustness and maturity of the
lovo-cel BLA package, expectations regarding the receipt of a
Priority Review Voucher upon potential approval of lovo-cel, and
bluebird bio’s ability to pursue curative gene therapies to give
patients and their families more bluebird days. 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, particularly those identified in
the risk factors discussion in bluebird bio’s Annual Report on Form
10-K for the year ended December 31, 2022, 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.
These risks include, but are not limited to: the risk that we may
not receive a Priority Review Voucher upon potential approval of
lovo-cel or that lovo-cel may not be approved in the timeframe we
anticipate or at all; we may encounter additional delays in the
development of our programs, including the imposition of new
clinical holds, which may impact our ability to meet our expected
timelines and increase our costs; the internal and external costs
required for our ongoing and planned activities, and the resulting
impact on expense and use of cash, has been and may in the future
be, higher than expected, which has caused us, and may in the
future cause us, to use cash more quickly than we expect or change
or curtail some of our plans or both; our expectations as to
expenses, cash usage and cash needs may prove not to be correct for
other reasons such as changes in plans or actual events being
different than our assumptions; the risk that the efficacy and
safety results from our prior and ongoing clinical trials will not
continue or be seen in additional patients treated with our product
candidates; the risk of insertional oncogenic or other reportable
events associated with lentiviral vector, drug product, or
myeloablation; and the risk that any one or more of our products or
product candidates, including lovo-cel, will not be successfully
developed, approved or commercialized. 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/20231030674385/en/
Investors & Media Investors: Courtney O’Leary,
978-621-7347 coleary@bluebirdbio.com
Media: Jess Rowlands, 857-299-6103
jess.rowlands@bluebirdbio.com
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