Refined commercial production spec gets gene therapy EMA go-ahead

US-based Bluebird Bio is working with German apceth Biopharma for commercial manufacturing of its one-time thalassemia therapy that is approved in the EU, not the US

Photo as seen on Apceth Biopharma website

The European Medicines Agency (EMA) has approved the refined commercial drug product manufacturing specifications for US-based Bluebird Bio, that ZYNTEGLO (autologous CD34+ cells encoding β A-T87Q-globin gene), a one-time gene therapy for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β00 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

"We look forward to serving TDT patients with ZYNTEGLO and providing a treatment option that offers the possibility of a transfusion-free future," said Alison Finger, Chief Commercial Office of Bluebird Bio. "This is one step along the commercial journey as we advance our ongoing launch and market access activities on a country-by-country basis, with the goal of enrolling our first commercial patient in 2019.

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 authorisation in the European Union. aZYNTEGLO addresses the underlying genetic cause of TDT and offers patients the potential to become transfusion independent, which once achieved is expected to be life-long.

"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," csaid Dr Christine Guenther, CEO of Apceth Biopharma. "We are proud to be the commercial manufacturing partner of Bluebird Bio and to be part of bringing this potentially life-changing therapy to TDT patients in Europe."

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