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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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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Pharmaceutical Technology

APRIL 13, 2023

Although the findings in the report are only preliminary, they shed light at the considerations behind the high price tags of gene therapies. Bluebird already uses a lentiviral vector in its approved gene therapy for beta-thalassemia called Zynteglo. Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.

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pharmaphorum

NOVEMBER 16, 2022

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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pharmaphorum

AUGUST 26, 2022

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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