First gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia who do not have a β0/β0 genotype now available to be manufactured in the
“We look forward to serving TDT patients with ZYNTEGLO and providing a treatment option that offers the possibility of a transfusion-free future,” said
The refined commercial drug product specifications support the efficacy and safety profile of ZYNTEGLO and will give patients the best opportunity for clinically meaningful outcomes consistent with the results that were foundational to the conditional marketing authorization in the
“These are exciting times also for apceth, as we are now in the final stages of preparing to manufacture a cell-based gene therapy for commercial use,” commented Dr.
Data Supporting Clinical Profile of ZYNTEGLO
The conditional marketing authorization is supported by efficacy, safety and durability data from the Phase 1/2 HGB-205 study and the completed Phase 1/2 Northstar (HGB-204) study as well as available data from the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies, and the long-term follow-up study LTF-303, as of the data cut off of
Non-serious adverse events (AEs) observed during clinical trials that were attributed to ZYNTEGLO were hot flush, dyspnoea, abdominal pain, pain in extremities and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to ZYNTEGLO.
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. At last follow up all patients treated with ZYNTEGLO in the clinical trial program remain alive.
For details, please see the Summary of Product Characteristics (SmPC). ZYNTEGLO has received a conditional marketing authorization in the
ZYNTEGLO continues to be evaluated in the ongoing Phase 3 Northstar-2 and Northstar-3 studies and the long-term follow-up study LTF-303.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent 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
ZYNTEGLO, LentiGlobin and Lenti-D 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.
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements the company’s commercialization plans and expectations for ZYNTEGLO. 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 we will encounter challenges in the commercial launch of ZYNTEGLO in the
Source: bluebird bio, Inc.