Bluebird Bio sets price of new gene therapy for β-thalassaemia at $1.8 million –– twice what analysts expected.

Transfusion-dependent β-thalassemia (TDT) is a rare genetic blood disorder caused by mutations in the gene that encodes β-globin, resulting in reduced or absent hemoglobin. The only current treatment is chronic blood transfusions, which carries the risk of progressive multi-organ damage due to unavoidable iron overload. Bluebird Bio received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use  in March 2019 and marketing approval at the end of May 2019 for Zynteglo (autologous CD34⁺ cells expressing β-[ A-T87Q]-globin), a gene therapy for patients 12 years and older with TDT who do not have a β[0]/β[0] genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is unavailable.

Recently, Bluebird announced its price for Zynteglo –– $1.8 million to be paid in installments over five years for patients who are successfully treated with the drug. That is twice what most analysts expected and makes Zynteglo the second most expensive therapy on the planet, behind Novartis’ newly approved gene therapy Zolgensma (onasemnogene abeparvovec-xioi), which is priced at $2.1 million.

Despite projections of $800 million in sales for TMT, with other indications in the pipeline including sickle cell diseases, Bluebird’s stock fell in response to the pricing news. The company says that the drug has an intrinsic value of $2.1 million, because it delivers 22 quality-adjusted life years for the most successful cases, so the price reflects a 15% discount. But Bluebird hasn’t yet proven that a one-time treatment is effective over a lifetime. To date, it has been shown to keep patients transfusion-free for nearly 4 years –– when successful.

It isn’t helping that Bluebird has delayed the launch of Zynteglo in order to make some last-minute changes to the manufacturing process in response to request amendments from the EMA. Any planned sales for 2019 will now be pushed to 2020. There is concern because, for cell and gene therapies, “the process is the product,” and Bluebird has not yet been able to deliver on the manufacturing process, from a regulatory perspective.