EU Panel Recommends Approving Gene Therapy for Thalassemia

Europe’s Committee for Medicinal Products for Human Use (CHMP) recommended approval of Bluebird Bio’s lentiglobin gene-therapy product for patients with transfusion-dependent beta-thalassemia. CHMP is the drug-reviewing arm of the European Medicines Agency, which is expected to issue a final decision on approval within the next three months.

The therapy involves obtaining mobilized autologous CD34-positive cells from patients, then transducing the cells ex vivo with the LentiGlobin BB305 vector, which encodes adult hemoglobin (HbA) with a T87Q amino-acid substitution (HbAT87Q). Patients undergo conditioning with busulfan, then modified cells are re-infused. In the New England Journal of Medicine, researchers reported that treatment with the lentiglobin therapy reduced or eliminated the need for long-term red blood cell transfusions in the majority of participants with severe, transfusion-dependent beta-thalassemia.

The CHMP reviewed data from a clinical trial of 10 patients with beta-thalassemia who required transfusions at baseline. After approximately three years of follow-up, eight patients were no longer receiving transfusions. The agency also reviewed data demonstrating that lentiglobin improved patients’ hemoglobin levels.

While exact pricing details are not yet available, if approved, the gene therapy is expected to carry a six-figure price tag. Bluebird Bio proposed a new installment-based and outcomes-based payment model earlier this year to allay potential concerns about the high cost of this one-time treatment.

Sources: STAT, March 29, 2019; Bluebird Bio press release, March 25, 2019.