Bluebird bio prices beta thalassaemia gene therapy Zynteglo at 1.575 million euros over five years

Bluebird bio on Friday proposed a five-year instalment plan for its gene therapy Zynteglo, with 315 000 euros ($354 000) to be paid up front and additional annual payments due only if the treatment continues to be effective, for a total of 1.575 million euros ($1.8 million). Zynteglo was conditionally approved in Europe earlier this month for certain patients 12 years and older with transfusion-dependent beta thalassaemia, and while the company did not disclose cost details at the time, it had previously suggested that the therapy would be priced below its "intrinsic value" of $2.1 million.

CEO Nick Leschly remarked that "the one-time potentially curative nature of what we have on our hands here sort of warrants this type of a [pricing] model in a more aggressive way." According to bluebird bio, the price is supported by the "expected lifelong clinical benefits" of Zynteglo, while the "payment model protects healthcare systems from bearing the cost of ineffective therapy."

The gene therapy, previously known as LentiGlobin, was awarded PRIME designation and orphan drug status by the European Medicines Agency for the treatment of transfusion-dependent beta thalassaemia. Bluebird bio explained that it is currently in talks with regulators in Germany, Italy, France and the UK regarding patient access, and that it intends to price Zynteglo in developed countries, including the US, within a "reasonably close" range.

Meanwhile, the company expects to receive approval for the treatment next year for use in patients with beta thalassaemia in the US, where it has been granted breakthrough therapy and orphan drug designations. Analysts anticipate Zynteglo to amass approximately $828 million in revenue by 2024.

Novartis recently announced that it would charge $2.1 million for its gene therapy Zolgensma (onasemnogene abeparvovac-xioi) following its US approval last month for paediatric patients with spinal muscular atrophy. The company also unveiled a plan to allow insurers to pay for Zolgensma over five years, averaging out to annual installments of $425 000. For related analysis, see ViewPoints: Novartis' Zolgensma sets the new status quo.

See also ViewPoints: bluebird gets greenlight for gene therapy in Europe and could soon put the company’s novel pricing strategy into play.  

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