SOMERVILLE, Mass.--(BUSINESS WIRE)--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.
