EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) confirms favorable benefit-risk balance of ZYNTEGLO
Company has informed EMA of lift of voluntary temporary marketing suspension
“Patient safety remains our top priority. To this end, we are grateful to the PRAC for its comprehensive review of the available evidence and positive recommendation for ZYNTEGLO,” said
No cases of hematologic malignancy have been reported in any patient who has received treatment with ZYNTEGLO. However because it is manufactured using the same BB305 lentiviral vector used in LentiGlobin for sickle cell disease (SCD; investigational drug product bb1111), bluebird bio decided to temporarily suspend marketing of ZYNTEGLO while the root cause of the safety events reported earlier this year for LentiGlobin for SCD were investigated by the company and assessed by the PRAC.
As previously announced on
The recommendation from the PRAC can be viewed here on the EMA website. As a next step, the recommendation will be forwarded to the Committee for Advanced Therapies (CAT) and Committee for Medicinal Products for Human Use (CHMP) for adoption. The final stage of the review procedure is the adoption by the
About ZYNTEGLO (betibeglogene autotemcel; beti-cel)
Betibeglogene autotemcel (beti-cel) is a one-time gene therapy that 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 adult hemoglobin (Hb), at levels that may eliminate or significantly reduce the need for transfusions. In studies of beti-cel, transfusion independence (TI) is defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.
beti-cel is manufactured using the BB305 lentiviral vector (LVV), a third-generation, self-inactivating LVV. The promoter, a regulatory element of the LVV that controls the expression of the transgene, selected for BB305 is a cellular (non-viral) promoter that drives gene expression only in the erythroid lineage cells (red blood cells and their precursors).
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. For details, please see the product information, containing Summary of Product Characteristics (SmPC).
bluebird bio is on track to complete its rolling Biologics License Application (BLA) submission to the FDA for beti-cel in mid-2021. This submission is anticipated to include adult, adolescent and children with transfusion dependent β-thalassemia across all genotypes (including non-β0/β0 genotypes and β0/β0 genotypes). Beti-cel is not approved in the
Beti-cel continues to be evaluated in the ongoing Phase 3
About bluebird bio, Inc.
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.
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ZYNTEGLO and bluebird bio are trademarks of bluebird bio, Inc.
bluebird bio Cautionary Statement Regarding 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 statements regarding the company’s expectations regarding the commercialization of ZYNTEGLO, and the timing of the submission of a BLA for beti-cel to the FDA. 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, which include but are not limited to: the risk that insertional oncogenic events associated with lentiviral vector or additional MDS events associated with transplant or myeloablation will be discovered or reported over time; the risk that insertional oncogenic events associated with lentiviral vector in other programs may result in a clinical hold of our programs in SCD, TDT or cerebral adrenoleukodystrophy; the risk that we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones, submissions or timelines, such as in the completion of our BLA submission for beti-cel and the renewal of the CMA for beti-cel; the risk that LentiGlobin for SCD or beti-cel will not be approved for marketing by the FDA, and the risk that we will not successfully bring LentiGlobin for SCD or beti-cel to market in
Source: bluebird bio, Inc.