FDA green light for beta thalassemia therapy Zynteglo

18 August 2022
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The US Food and Drug Administration approved Zynteglo (betibeglogene autotemcel), also known as beti-cel, from bluebird bio (Nasdaq: BLUE) the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

News of the much anticipated FDA decision pushed bluebird’s shares up more than 15% to $7.80 in pre-market trading this morning.

“As the first and only FDA-approved gene therapy for people with beta-thalassemia who require regular red blood cell transfusions, and with the potential to untether patients from a lifelong, burdensome treatment regimen, Zynteglo is setting the standard for what a one-time therapy can deliver,” said Tom Klima, chief commercial and operating officer, bluebird bio. “We have developed an equally innovative commercial infrastructure in collaboration with payers and providers, and with a focus on patients, to enable broad and timely access to Zynteglo for eligible patients and to support patients and families at every step of the treatment journey,” he added.

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