Bluebird Confident Beti-Cel Will Fare Better In US Than Europe

 
• By Alaric DeArment
  
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The company built on its case for FDA approval with new beta-thalassemia data at ASH, but investor jitters linger after the pullout of Zynteglo from Europe amid reimbursement woes for the gene therapy.

Beta thalassemia blood sample - Image
bluebird bio presented data for beti-cel in beta thalassemia at ASH • Source: Shutterstock

The data that bluebird bio presented at the American Society of Hematology meeting for betibeglogene autotemcel (beti-cel) certainly bolster the gene therapy’s case for US Food and Drug Administration approval for transfusion-dependent beta-thalassemia (TDT). However, the question is how effectively the company can execute on beti-cel’s commercialization in the US after pulling out of Europe due to difficulty securing reimbursement there.

The company presented data on 11 December at the ASH meeting from the LTF-303 long-term follow-up study, which includes patients...

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