The European Commission (EC) gave bluebird bio’s gene therapy, Zyntelgo (autologous CD34+ cells encoding βA-T87Q-globin gene), conditional marketing authorisation based on data from completed Phase I/II trials and an ongoing Phase III trial.
The treatment is approved for patients 12 years and older with transfusion-dependent β-thalassemia (TDT) who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen-matched related HSC donor is not available.
TDT is caused by a mutation of the β-globin gene resulting in reduced or no haemoglobin being produced by the body, which Zyntelgo treats the patient by adding functional copies of a modified form of the β-globin gene.
According to bluebird, the effects of the treatment are expected to last for the patient’s lifetime, which would negate the need for them to undergo repeated blood transfusions for the rest of their lives.
‘Chief bluebird’, Nick Leschly, explained, “Our first product approval is a humbling moment for all of us at bluebird, and we look forward to continuing our work with the TDT community and health systems to bring this important treatment to patients.”
The company’s first approval means the treatment will be available across all European Union (EU) countries, as well as Iceland, Liechtenstein, and Norway.
Bluebird noted that the approval was the fastest assessment of an advanced therapy medicinal product by the European Medicines Agency (EMA), following an accelerated assessment.
For bluebird, it represents the first stage in what could become a succession of treatments reaching the market, with Lenti-D and its chimeric antigen receptor (CAR)-T treatment, bb2121, edging closer to approval.
A question of pricing
At the end of last month, Novartis entered the market with gene therapy treatment Zolgensma (onasemnogene abeparvovec), which promptly became the most expensive treatment on the market.
At $2.1m (€1.87m) for the one-time treatment, the sustainability of this kind of pricing was raised, and bluebird will likely face similar questions should it price its own treatment at a similar level.
Leschly has previously suggested that a reasonable price would be $2.1m for the one time therapy, which would be spread over a five-year period.