Pharmaceutical Technology

SEPTEMBER 30, 2022

Last month, Zynteglo (betibeglogene autotemcel), or beti-cel, was approved as a one-time potentially curative gene therapy for patients with beta-thalassaemia who require regular blood transfusions. After several setbacks, bluebird bio bounces back with two major FDA gene therapy approvals.

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pharmaphorum

AUGUST 18, 2022

bluebird bio’s Zynteglo has become the first cell-based gene therapy to be approved in the US, getting the nod from the FDA as a treatment for patients with beta thalassaemia who require regular blood transfusions. An FDA advisory committee recommended approval of Zynteglo on the strength of that data in June.

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STAT

OCTOBER 6, 2022

million tag as cost effective for a Bluebird Bio drug called Zynteglo for the blood disorder beta thalassemia, Arbuckle said in an interview.  Even a drug-price watchdog, the Institute for Clinical and Economic Review, deemed the $2.8

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pharmaphorum

FEBRUARY 22, 2021

Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. . The post Zynteglo halt re-ignites viral vector safety concerns; analysts appeared first on.

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Pharmaceutical Technology

NOVEMBER 9, 2022

Reimbursement has been a major obstacle in the EU for expensive gene therapies, including bluebird bio’s beta-thalassemia therapy Zynteglo. The European Commission withdrew the marketing authorisation of Zynteglo, at the request of bluebird bio, in March 2022. After its approval, bluebird had set Zynteglo’s price at roughly EUR1.6

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pharmaphorum

APRIL 14, 2022

million price tag. The FDA is currently reviewing beti-cel – also known as Zynteglo (betibeglogene autotemcel) – as a treatment for patients with beta thalassaemia who require regular blood transfusions.

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pharmaphorum

JUNE 12, 2022

Beti-cel has already been approved for marketing in Europe as Zynteglo, with a price tag of around $1.8 million price tag, saying its ability to help patients reach sustained transfusion independence justified its high price. Beti-cel offers another potentially curative treatment for a much broader range of patients.

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