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On June 3, European regulators gave a green light to bluebird bio's (NASDAQ: BLUE) beta-thalassemia therapy, Zynteglo, and on Friday, management revealed plans to charge $1.8 million for the revolutionary new treatment. Its million-plus price sounds exorbitant, but it may not be as ridiculous as it seems. Here's how Zynteglo may transform the treatment of this genetic disorder.
What's beta-thalassemia?
It's a potentially fatal disease. Beta-thalassemia patients can't produce adequate levels of a protein called beta-globin because of a genetic mutation. Beta-globin is a component of hemoglobin, an iron-rich protein in red blood cells responsible for transporting oxygen from the lungs to the body's tissues. In severe cases of beta-thalassemia, patients require red blood cell transfusions every two to five weeks in order to survive.
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These transfusions can help patients avoid severe anemia that can lead to organ damage, but they're expensive, and over time, they can result in iron overload that can be life-threatening.
A game-changing new approach
A lifetime of blood transfusions may no longer be necessary for many following the European approval of bluebird bio's Zynteglo. A gene therapy, Zynteglo inserts a functional copy of a modified form of the beta-globin gene (βA-T87Q-globin gene) into a patient's own blood stem cells.
The new gene allows patients to produce their own beta-globin, potentially eliminating the need for blood transfusions.
In bluebird bio's HGB-205 study, three out of four patients without the β0/β0 genotype of this disease achieved transfusion independence lasting at least 12 months following one dose of Zynteglo. In its Northstar study, eight of 10 patients lacking the β0/β0 genotype were transfusion-independent for 21 to 56 months post-treatment.
Those results were good enough to secure European approval for use in transfusion-dependent patients over the age of 12 who don't have the β0/β0 genotype, but trials could expand use to people with that genotype in the future.
Is $1.8 million ridiculous?
Gene therapies like Zynteglo that restore protein expression and remove the need for chronic treatment could actually save healthcare systems a lot of money over time, despite their million-dollar price tags.
Earlier this year, bluebird bio presented an analysis demonstrating Zynteglo's ability to improve quality of life and extend life expectancy gives it an intrinsic value of $2.1 million. At the time, the company pledged to price it below that figure and to entertain novel payment terms, including deals that spread payments out over several years.