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- BLAs for beti-cel for beta-thalassemia and eli-cel for cerebral adrenoleukodystrophy, to be discussed at an FDA Advisory Committee Meeting on June 9-10, 2022 -
SOMERVILLE, Mass., June 09, 2022--(BUSINESS WIRE)--bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") today announced that Nasdaq has halted trading of the company’s common stock on Thursday, June 9 and Friday, June 10, 2022. The U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) is meeting to discuss the biologics licensing applications (BLAs) for betibeglogene autotemcel (beti-cel) and elivaldogene autotemcel (eli-cel).
Beti-cel is under review for the treatment of people with β-thalassemia who require regular red blood cell transfusions. Eli-cel is under review for the treatment of early active cerebral adrenoleukodystrophy (CALD) in patients less than 18 years of age who do not have an available and willing human leukocyte antigen (HLA)-matched sibling hematopoietic stem cell (HSC) donor.
Briefing materials, agendas and webcast information for the meeting can be accessed here. The Company is not responsible for the content of, nor the statements made in, the briefing materials that were prepared by the FDA.
The Prescription Drug User Fee Act (PDUFA) goal dates for a decision on approval of beti-cel for people with beta-thalassemia who require regular red blood cell transfusions and eli-cel for patients with early active CALD without a matched sibling donor are August 19, 2022, and September 16, 2022, respectively.
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 clinical programs for sickle cell disease, beta-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.
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 Company’s plans and expectations for anticipated FDA approval of the BLAs for beti-cel and eli-cel. Such forward-looking statements are based on historical performance and current expectations about our future 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, 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: we may encounter additional delays in the development of our programs, including the imposition of new clinical holds or delays in resolving existing clinical holds, that may impact our ability to meet our expected timelines; 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 that additional insertional oncogenic or other reportable events associated with lentiviral vector, drug product, or myeloablation will be discovered or reported over time; the risk that our eli-cel, beti-cel and lovo-cel programs may be subject to further delays in their development, including but not limited to the imposition of new clinical holds; the risk that eli-cel and/or beti-cel may not be approved within the priority review timeframe or at all; the risk that any one or more of our product candidates, including eli-cel and/or beti-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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220609005538/en/
Courtney O’Leary, 978-621-7347
Jess Rowlands, 857-299-6103