bluebird bio’s Zynteglo Approved by the FDA for Beta-Thalassemia

Anna Osborne
24 August 2022
Gene Therapy
Patient Access & Engagement
Regulatory & Standards
Viral/Non-Viral Vectors
bluebird bio has announced that the FDA has approved its gene therapy Zynteglo, otherwise known as beti-cel, for the treatment of beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.

The FDA’s approval of Zynteglo is based on data from bluebird’s multiple Phase III clinical trials, which were part of the longest and most robust clinical gene therapy program in transfusion-dependent beta-thalassemia (TDT).

Results from these Phase III studies demonstrated that 89% of the patients eligible for evaluation achieved transfusion independence – they maintained a weighted average total hemoglobin of at least 9 g/dL and therefore no longer needed RBC transfusions for at least 12 months post-therapy.

bluebird also report that all those who were reported to achieve transfusion independence have remained transfusion free at the last follow-up.

A long-term 15-year follow-up study monitoring safety and efficacy in the program populations is also currently being conducted by bluebird.

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Zynteglo is a one-time ex-vivo lentiviral vector gene therapy offering potentially curative benefits to those living with TDT. The single-dose nature of this therapy, and the clinical evidence of transfusion independence, provides hope for an improved quality of life for those with this debilitating disease.

To date, the current standard of care for people living with TDT involves transfusions up to every two to five weeks. This can be significantly disruptive to the lives of those receiving the transfusion, as well as their caregivers and families.

Additionally, these patients receive daily therapies and monitoring to limit the risk of iron overload and potentially fatal complications.

The lifetime medical costs for the approximately 1,500 people in the U.S. living with this form of beta-thalassemia can reach up to $6.4 million per patient – 23 times higher than the average of the general population.

Zynteglo’s wholesale acquisition cost is set at $2.8 million, reflecting its clinically proven potential to alleviate the long-term costs of frequent RBC transfusions and iron management in the beta-thalassemia patient population.


A bluebird Company representative explains that the cost to payers of Zynteglo is published after formulation by Company experts using a specific methodology. Included in these calculations are considerations of the broader benefits to society this therapy can offer. For example, the workforce contribution caregivers and family members of those with TDT could add given reduced hospital trips for transfusions.

In recognition of the large price tag that comes with Zynteglo bluebird is committed to finding flexible strategies to meet payer’s needs, including out-come-based agreements. One-off payments are coupled with a contract of reimbursement of 80% if the patient fails to maintain transfusion independence at the two-year follow-up.

Zynteglo will be available to those living with the TDT through bluebird Qualified Treatment Centers (QTCs) across the U.S., located in leading healthcare centers chosen for their expertise in beta-thalassemia and cell and gene therapies.

The first of the QTC network are to be trained and activated this September in time for bluebird’s plans for the first patient apheresis by the end of 2022.

Those living with transfusion-dependent beta-thalassemia in the U.S. will also have access to bluebirds’ new initiative ‘my bluebird support’. This program will connect those considering Zynteglo, and their families, with Patient Navigators to provide support, education, and advice as they embark on their gene therapy journey.

“I’m delighted for bluebird bio and more so for people living with beta-thalassemia in the USA, it’s brilliant to think this treatment can soon become a choice for them,” comments Nicola Redfern, Independent Consultant at NJ Redfern Ltd and previous GM for bluebird bio in the UK.

Considering this news from the perspective of the European cell and gene therapy industry Nicola adds, “At the same time, it is also devastating that it isn’t available in Europe and the UK. It is not unusual for there to be differences and often a delay in therapeutic approval from the US to the European market – but this situation is different given bluebird’s recent departure from the European market. bluebird was very proactive to try and engage with payers across multiple countries in Europe to find solutions to bring this to market quickly but sadly it was really difficult and not successful. There was a significant chasm between what the payers were prepared to pay and what bluebird needed to be a viable business across Europe. I imagine that any hope of replicating the success of Zynteglo in the US here at a future point, and bluebird bio coming back, would take a proactive indication from multiple countries across Europe that there was a strong appetite to reach an agreement, and reach it quickly.”

“Let’s not wait another 10 years before access to such life-saving therapies becomes a reality for more families,” she concludes.

Source: bluebird bio Press Release

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