Bluebird’s ‘one-time gene therapy’ receives EMA accelerated assessment

By Ben Hargreaves contact

- Last updated on GMT

(Image: Getty/Royyimzy)
(Image: Getty/Royyimzy)

Related tags: bluebird bio, CAR-T, Gene therapy

Bluebird bio’s LentiGlobin gene therapy has received an accelerated assessment from the EMA for the treatment of transfusion-dependent β-thalassemia.

The European Medicines Agency’s (EMA) nod for bluebird bio, ahead of its marketing authorisation application (MAA), follows on from the PRIME designation​ provided to the therapy in September 2016.

LentiGlobin has the potential to be a one-time treatment that could address the genetic cause of transfusion-dependent β-thalassemia (TDT) in patients with a non-β0/β0 genotype.

TDT is an inherited blood disorder caused by a mutation in the beta-globin gene, which results in an insufficient number of healthy red blood cells and the need for regular blood transfusions.

Chief medical officer of bluebird bio, David Davidson, explained how these transfusions place a burden on patients’ lives: TDT requires a lifetime of chronic blood transfusions for survival. These blood transfusions are life-saving, but they are also associated with serious medical complications, such as organ failure from iron overload. In addition to medical complications, many patients find that managing transfusions impacts their personal and professional lives.”

Davidson explained to us that the hope for LentiGlobin is that it could remove the need for transfusions, or, at least, reduce patients’ reliance on them.

Such an innovation would make patients’ lives easier and also reduce costs to the healthcare system.

However, as with other breakthroughs​ in the cell and gene therapy area, the question of pricing is a significant one; with its own work in the CAR-T space​, it’s a conversation that bluebird bio is familiar with.

When questioned on what the pricing structure would be, should the treatment receive eventual approval, a spokesperson for bluebird bio commented: Our gene therapy treatments are delivered once – but have the potential to deliver benefit over a patient’s lifetime.

“With that said, we recognize there is not a one-size-fits-all approach to pricing. For some payers, the best approach may involve an annuity-based or pay-for-performance model, while this type of model may not work for others. We plan to continue work with stakeholders to discuss potential pricing models.”

The spokesperson confirmed that bluebird plans to file an MAA before the end of the year.

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