Patients with rare blood disorder benefit from Bluebird gene therapy while denied access in Europe

People with severe beta-thalassemia who live in Europe have been denied access to Bluebird Bio’s approved gene therapy after the company withdrew it from the market last April. European health systems balked at the nearly $2 million price tag for the gene therapy called Zynteglo, and when negotiations broke down, Bluebird walked away.

But Saturday, Bluebird relied largely on European patients enrolled in its beta-thalassemia clinical trials to demonstrate the successful, long-term durability of Zynteglo. The juxtaposition is awkward for Bluebird, showing business priorities don’t always align with the biotech’s “patients first” mantra.

More than 80% of severe beta-thalassemia patients treated with Zynteglo remain transfusion-independent after five years of follow up. A few patients continue to benefit for up to seven years, according to pooled data from multiple clinical trials presented by Bluebird at the annual meeting of the American Society of Hematology.

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