Remove tag thalassaemia
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bluebird bio wins back-to-back landmark FDA approvals for first-in-class gene therapies

Pharmaceutical Technology

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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bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval

pharmaphorum

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. bluebird has opted to launch it at a cost of $2.8 million, which compares to the $1.7 million, spit between $1.6

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Advanced therapies and the high-profile pricing dilemma

pharmaphorum

Only a few years later, bluebird bio’s gene therapy, Zynteglo (betibeglogene autotemcel), was approved in the European market for the treatment of the rare blood disorder, beta thalassaemia. However, with a price tag of €1.58 At more than $2.1 The post Advanced therapies and the high-profile pricing dilemma appeared first on.

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Roctavian okay sets up another gene therapy test for Europe

pharmaphorum

BioMarin will no doubt have an eye on the earlier experience of bluebird bio, which secured EU approval for rare disease gene therapy Zynteglo (betibeglogene autotemcel) in 2019 for beta thalassaemia with a price tag of around €1.6 million spread over five years.

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