Bluebird Bio Prices TDT Gene Therapy at $1.8 Million

Bluebird Bio said it will charge $1.78 million over five years for Zynteglo, its gene therapy to treat transfusion-dependent beta thalassemia (TDT), a rare genetic disorder that results in reduced or absent hemoglobin essential for transporting oxygen within the body.

Photo: Nick Leschly, CEO of Bluebird Bio

The pricing of Zynteglo places it just behind Novartis’ gene therapy Zolgesma, which is priced at $2.1 million over five years.

Zynteglo recently received conditional approval in the European Union for the treatment of patients 12 years and older with TDT. In order to survive, people with TDT maintain hemoglobin levels through lifelong chronic blood transfusions. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload. Zynteglo was approved for those patients who do not have the ß0/ ß0 genotype and for whom there is no matching stem cell transplant donor.

Bluebird announced the price for Zynteglo on a webcast during the European Hematology Association Congress in Amsterdam, where CEO Nick Leschly made the case that Zynteglo has an intrinsic value of $2.1 million because it delivers 22 quality-adjusted life years for patients for whom the gene therapy is successful, and noted that Zynteglo is priced at a 15 percent discount to that figure.

Patients will pay $356,567 per year for five years as long as the gene therapy works for them. It remains to be seen if the new payment models for one-time gene therapies will work as there is yet no definitive proof of the gene therapy’s durability.

During the webcast and at EHA, Bluebird presented long-term efficacy and safety data from ongoing clinical studies of Zynteglo that showed that about 80 percent of patients receiving the gene therapy (4 out of 5 in a study of patients with a severe form of TDT, and 11 out of 14 in another) achieved transfusion independence, for anywhere from three months to 3.8 years for one patient with a milder form of the disease. But in another group of eight patients with the ß0/ ß0 genotype, only three were transfusion independent.

Bluebird also said that it is working with the European Medicines Agency to finalize commercial drug product specifications and manufacturing parameters, which will likely push the commercial launch of Zynteglo to the end of 2019 or early 2020.